UKDP: Integrated DEmentiA research environment (IDEA)
Lead Research Organisation:
University College London
Department Name: Institute of Neurology
Abstract
The UK Dementias Platform (UKDP) is a radically new approach to dementias research. It brings together data from
around 2,000,000 study participants from 22 cohorts to try and discover the causes of dementia and to find out ways of
slowing it down. The platform has been funded to the level of £12M.
In this proposal we want to improve UK infrastructure for dementia research so that the most can be made of the
opportunity provided by UKDP. We wan to improve the infrastructure by buying important pieces of equipment that will put
the UK at the forefront of dementia research worldwide and by establishing networks of scientists who will work together to
make best use of the equipment.
The three themes of the proposal are imaging, informatics and stem cells.
For imaging we want to establish a network of PET/MR scanning facilities across the UK so that the molecular processes
going on inside the brain that cause dementia can be studied.
For informatics we want to be bring together bas many different types of data as possible and make it easy as possible for
scientists to use them
For stem cells we want to take cells form adults with and without dementia to find out how cells change as the dementias
process begins and progresses.
We believe these proposals will raise standards, reduce costs, and deliver innovative and coordinated research, making
the UK an internationally unique place to study dementia.
around 2,000,000 study participants from 22 cohorts to try and discover the causes of dementia and to find out ways of
slowing it down. The platform has been funded to the level of £12M.
In this proposal we want to improve UK infrastructure for dementia research so that the most can be made of the
opportunity provided by UKDP. We wan to improve the infrastructure by buying important pieces of equipment that will put
the UK at the forefront of dementia research worldwide and by establishing networks of scientists who will work together to
make best use of the equipment.
The three themes of the proposal are imaging, informatics and stem cells.
For imaging we want to establish a network of PET/MR scanning facilities across the UK so that the molecular processes
going on inside the brain that cause dementia can be studied.
For informatics we want to be bring together bas many different types of data as possible and make it easy as possible for
scientists to use them
For stem cells we want to take cells form adults with and without dementia to find out how cells change as the dementias
process begins and progresses.
We believe these proposals will raise standards, reduce costs, and deliver innovative and coordinated research, making
the UK an internationally unique place to study dementia.
Technical Summary
Our objective is to achieve a step-change in UK dementia research capacity through establishing national networks of
existing and emerging centres of excellence in imaging, informatics and cell-biology.
The UK Dementias Platform (UKDP) is a radically new approach to dementias research, providing a highly efficient and
cost-effective translational pipeline from discovery through to early phase trials. UKDP will create closer synergy between
epidemiology and experimental medicine with the re-purposing of epidemiologic cohorts for trials readiness. The size and
depth of phenotyping available to UKDP will deliver a step-change in the complexity and granularity of dementia related
hypothesis testing and accelerate compound development.
Proposed here is an infrastructure of investment and collaboration. Underpinning UKDP is a critical mass of researchers
and resources that will work together to encourage, facilitate, and develop a fully integrated dementia dedicated UK
research environment. This will raise standards, reduce costs, and deliver innovative and coordinated research, to make
the UK an internationally unique research environment. Through its partnership with major academic centres and industry,
UKDP is well positioned to achieve this goal.
Building on the recent MRC investment in UKDP, we propose here to renew and extend the UKDP integrative research
environment with an advanced molecular imaging network strategically located to exploit UKDP cohorts. Also proposed is
an integrated informatics environment to facilitate the location of and access to both data and bio-samples. The third
proposal is for a stem-cells network to promote the use of this important and emerging technology. Each of these elements
adds value to existing infrastructure investments and fills significant gaps in the UK research landscape.
existing and emerging centres of excellence in imaging, informatics and cell-biology.
The UK Dementias Platform (UKDP) is a radically new approach to dementias research, providing a highly efficient and
cost-effective translational pipeline from discovery through to early phase trials. UKDP will create closer synergy between
epidemiology and experimental medicine with the re-purposing of epidemiologic cohorts for trials readiness. The size and
depth of phenotyping available to UKDP will deliver a step-change in the complexity and granularity of dementia related
hypothesis testing and accelerate compound development.
Proposed here is an infrastructure of investment and collaboration. Underpinning UKDP is a critical mass of researchers
and resources that will work together to encourage, facilitate, and develop a fully integrated dementia dedicated UK
research environment. This will raise standards, reduce costs, and deliver innovative and coordinated research, to make
the UK an internationally unique research environment. Through its partnership with major academic centres and industry,
UKDP is well positioned to achieve this goal.
Building on the recent MRC investment in UKDP, we propose here to renew and extend the UKDP integrative research
environment with an advanced molecular imaging network strategically located to exploit UKDP cohorts. Also proposed is
an integrated informatics environment to facilitate the location of and access to both data and bio-samples. The third
proposal is for a stem-cells network to promote the use of this important and emerging technology. Each of these elements
adds value to existing infrastructure investments and fills significant gaps in the UK research landscape.
Planned Impact
The current proposal in in support of the UKDP and will become part of the the UKDP impact strategy.
In summary, the UKDP strategy to deliver pact is to develop networks of partnership to actively consult engage the UK
academic community in relation to dementia research focussing on the direction,
technologies and collaborations of the UKDP and the wider UK national infrastructure.
Our aim is to:
1) promote the best possible science
2) create momentum in dementias research by being inclusive of, and synergistic with, other initiatives.
Partnership discussions with industry are already underway with exchanges of ides, interests and needs between
academic and industry stakeholders. Industry have identified their need for access to conversion (early MVI to dementia
cohorts and for experimental medicine studies to conform to regulatory requirements.
We remain committed to raising the profile of contemporary debate about dementia and its treatment. We wish to
encourage a culture of commitment to solving this problem. By increasing awareness at all levels of society we intend to
leverage resources for the platform and for dementia research in general, to increase awareness of the need for earlier
interventions and better targeted treatment in general by health service providers and the public alike.
Engagement with the general public and with patients and carers is a very important part of our mission. This serves not
only to communicate our research findings and their relevance but also to address such issues as stigma in society and the
research culture in the NHS in relation to dementia and older people. In addition to using the platform web-site to
communicate to the general public, we will also liaise with charities and advocate groups such as Age UK and the
Alzheimer's Society to promote our work and findings and to engage them in shaping the work programme.
In addition we will have a dedicated free-phone number available 6 days a week and a communications officer at 50% FTE
over 5 years whose responsibility is to develop and implement a communications and public engagement strategy.
In summary, the UKDP strategy to deliver pact is to develop networks of partnership to actively consult engage the UK
academic community in relation to dementia research focussing on the direction,
technologies and collaborations of the UKDP and the wider UK national infrastructure.
Our aim is to:
1) promote the best possible science
2) create momentum in dementias research by being inclusive of, and synergistic with, other initiatives.
Partnership discussions with industry are already underway with exchanges of ides, interests and needs between
academic and industry stakeholders. Industry have identified their need for access to conversion (early MVI to dementia
cohorts and for experimental medicine studies to conform to regulatory requirements.
We remain committed to raising the profile of contemporary debate about dementia and its treatment. We wish to
encourage a culture of commitment to solving this problem. By increasing awareness at all levels of society we intend to
leverage resources for the platform and for dementia research in general, to increase awareness of the need for earlier
interventions and better targeted treatment in general by health service providers and the public alike.
Engagement with the general public and with patients and carers is a very important part of our mission. This serves not
only to communicate our research findings and their relevance but also to address such issues as stigma in society and the
research culture in the NHS in relation to dementia and older people. In addition to using the platform web-site to
communicate to the general public, we will also liaise with charities and advocate groups such as Age UK and the
Alzheimer's Society to promote our work and findings and to engage them in shaping the work programme.
In addition we will have a dedicated free-phone number available 6 days a week and a communications officer at 50% FTE
over 5 years whose responsibility is to develop and implement a communications and public engagement strategy.
Organisations
- University College London (Lead Research Organisation)
- Francis Crick Institute (Collaboration)
- University College London (Collaboration)
- University of Manchester (Collaboration)
- Alzheimer's Research UK (Collaboration)
- UNIVERSITY OF CAMBRIDGE (Collaboration)
- Aligning Sciences Across Parkinson's (Collaboration)
- UNIVERSITY OF OXFORD (Collaboration)
- UNIVERSITY OF EDINBURGH (Collaboration)
- AstraZeneca (Collaboration)
- Dementia Discovery Fund (Collaboration)
- Janssen Pharmaceutica NV (Collaboration)
- Cardiff University (Collaboration)
- National Institute of Allergy and Infectious Diseases (NIAID) (Collaboration)
- Karolinska University Hospital (Collaboration)
- Stanford University (Collaboration)
- University of Antwerp (Collaboration)
- Perkin Elmer (Collaboration)
- Cerevance Ltd (Collaboration)
Publications
Lee DDH
(2021)
Higher throughput drug screening for rare respiratory diseases: readthrough therapy in primary ciliary dyskinesia.
in The European respiratory journal
Lopes Da Silva M
(2016)
Type II PI4-kinases control Weibel-Palade body biogenesis and von Willebrand factor structure in human endothelial cells.
in Journal of cell science
Lopes-Da-Silva M
(2019)
A GBF1-Dependent Mechanism for Environmentally Responsive Regulation of ER-Golgi Transport.
in Developmental cell
Luisier R
(2018)
Intron retention and nuclear loss of SFPQ are molecular hallmarks of ALS
in Nature Communications
Mamais A
(2018)
Analysis of macroautophagy related proteins in G2019S LRRK2 Parkinson's disease brains with Lewy body pathology.
in Brain research
Martinez-Miguel VE
(2021)
Increased fidelity of protein synthesis extends lifespan.
in Cell metabolism
Mauricio R
(2019)
Tackling gaps in developing life-changing treatments for dementia.
in Alzheimer's & dementia (New York, N. Y.)
Mazzon M
(2019)
Identification of Broad-Spectrum Antiviral Compounds by Targeting Viral Entry.
in Viruses
McCormack JJ
(2020)
Human endothelial cells size-select their secretory granules for exocytosis to modulate their functional output.
in Journal of thrombosis and haemostasis : JTH
Title | magazine cover single molecule microscopy |
Description | Cover art for Journal Chemical Biology Biological Chemistry 2018 |
Type Of Art | Artwork |
Year Produced | 2018 |
Impact | Increased awareness of TAB amyloid microscopy as a novel research tool; promotion of science in the public sphere |
URL | http://doi.org/10.1002/cbic.201800489 |
Description | International 3Rs Prize 2018 - please see awards section for details of impact |
Geographic Reach | National |
Policy Influence Type | Influenced training of practitioners or researchers |
Description | Member of expert panel advising the Dementia Discovery Fund for potential investments in autophagy research |
Geographic Reach | Multiple continents/international |
Policy Influence Type | Participation in a guidance/advisory committee |
Description | Advancing the GLP-1 receptor as a target in Parkinson's disease |
Amount | £68,000 (GBP) |
Organisation | Michael J Fox Foundation |
Sector | Charity/Non Profit |
Country | United States |
Start | 09/2018 |
End | 10/2019 |
Description | Apollo Therapeutics |
Amount | £732,918 (GBP) |
Organisation | Apollo Therapeutics |
Sector | Private |
Country | United Kingdom |
Start | 01/2020 |
End | 12/2021 |
Description | CBD Solutions Research Grant |
Amount | $480,000 (USD) |
Organisation | Karin & Sten Mortstedt CBD Solutions AB |
Sector | Private |
Country | Sweden |
Start | 06/2018 |
End | 12/2020 |
Description | CHDI Foundation grant: Mechanism of the DNA damage response in Huntington's disease pathogenesis |
Amount | £753,654 (GBP) |
Organisation | CHDI Foundation |
Sector | Charity/Non Profit |
Country | United States |
Start | 05/2018 |
End | 05/2021 |
Description | CRUK City of London Centre Radiation Research Unit |
Amount | £10,533,064 (GBP) |
Funding ID | 28990 |
Organisation | Cancer Research UK |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 11/2019 |
End | 10/2024 |
Description | Capital Equipment Fund, CEF3 |
Amount | £500,000 (GBP) |
Organisation | University College London |
Sector | Academic/University |
Country | United Kingdom |
Start | 07/2019 |
End | 07/2020 |
Description | Dissecting the mechanisms underlying disease progression in parkinsonism |
Amount | $7,014,337 (USD) |
Organisation | Aligning Sciences Across Parkinson's |
Sector | Charity/Non Profit |
Country | United States |
Start | 01/2021 |
End | 12/2023 |
Description | Dissecting the mechanisms underlying disease progression in parkinsonism |
Amount | £7,014,337 (GBP) |
Funding ID | ASAP-000478 |
Organisation | Aligning Sciences Across Parkinson's |
Sector | Charity/Non Profit |
Country | United States |
Start | 03/2021 |
End | 03/2024 |
Description | EU Innovative Medicines Initiative |
Amount | € 900,000 (EUR) |
Organisation | European Commission |
Sector | Public |
Country | European Union (EU) |
Start | 05/2016 |
End | 05/2021 |
Description | Eisai-UCL Collaboration Project TIG E |
Amount | £231,000 (GBP) |
Organisation | Eisai Ltd |
Sector | Private |
Country | Japan |
Start | 11/2017 |
End | 04/2020 |
Description | Elucidating early stage ALS pathomecanisms that drive mitochondrial dysfunction |
Amount | £1,046,186 (GBP) |
Funding ID | MR/S025898/1 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 01/2020 |
End | 12/2023 |
Description | Enhanced mitophagy as therapeutic opportunity in Parkinsons disease |
Amount | £256,201 (GBP) |
Funding ID | TIG F |
Organisation | Eisai Ltd |
Sector | Private |
Country | Japan |
Start | 04/2020 |
End | 04/2022 |
Description | Exploring the molecular mechanisms of KAT8/KANSL1-dependent regulation of mitophagy |
Amount | $193,744 (USD) |
Organisation | Michael J Fox Foundation |
Sector | Charity/Non Profit |
Country | United States |
Start | 06/2020 |
End | 01/2022 |
Description | Exploring the molecular mechanisms of KAT8/KANSL1-dependent regulation of mitophagy |
Amount | $19,374,424 (USD) |
Organisation | Michael J Fox Foundation |
Sector | Charity/Non Profit |
Country | United States |
Start | 03/2020 |
End | 08/2021 |
Description | High Content Screening (HCS) platform for HD drug discovery |
Amount | £381,185 (GBP) |
Organisation | Takeda Pharmaceutical Company |
Sector | Private |
Country | Japan |
Start | 12/2017 |
End | 12/2019 |
Description | Investigating proteostasis in development and disease using iPSC neurons with MAPT mutations linked to FTD |
Amount | £107,034 (GBP) |
Funding ID | BB/S506886/1 |
Organisation | Biotechnology and Biological Sciences Research Council (BBSRC) |
Sector | Public |
Country | United Kingdom |
Start | 08/2018 |
End | 09/2022 |
Description | Lysosome Function Enhancement to Treat Synucleinopathies |
Amount | £384,000 (GBP) |
Funding ID | TIG G |
Organisation | Eisai Ltd |
Sector | Private |
Country | Japan |
Start | 03/2020 |
End | 03/2022 |
Description | Lysosome turnover in health, aging and disease |
Amount | £1,232,805 (GBP) |
Funding ID | 212216/Z/18/Z |
Organisation | Wellcome Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 05/2019 |
End | 05/2024 |
Description | MRC Partnership Grant |
Amount | £1,220,774 (GBP) |
Funding ID | MR/N013255/1 |
Organisation | Medical Research Council (MRC) |
Sector | Public |
Country | United Kingdom |
Start | 05/2016 |
End | 05/2019 |
Description | Mechanism of the DNA damage response in Huntington's disease pathogenesis |
Amount | £1,574,978 (GBP) |
Funding ID | A-15806 |
Organisation | CHDI Foundation |
Sector | Charity/Non Profit |
Country | United States |
Start | 08/2020 |
End | 09/2022 |
Description | Modeling Parkinson's disease in 2D and 3D. |
Amount | £82,300 (GBP) |
Organisation | MRC Doctoral Training Program |
Sector | Academic/University |
Country | United Kingdom |
Start | 09/2018 |
End | 10/2021 |
Description | Molecular Characterization of FAN1 Variation in Huntington's disease |
Amount | £163,169 (GBP) |
Organisation | CHDI Foundation |
Sector | Charity/Non Profit |
Country | United States |
Start | 01/2017 |
End | 06/2018 |
Description | Non cell autonomous Alzheimer's disease in iPSC derived cells - astrocyte reactivity and APP signalling |
Amount | £224,932 (GBP) |
Funding ID | 177986 |
Organisation | Alzheimer's Society |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 12/2018 |
End | 02/2022 |
Description | RElapse-specific therapeutic Vulnerability Evaluation in childhood & young adult ALL (REVEALL) |
Amount | £968,000 (GBP) |
Funding ID | DRCPGM\100066 |
Organisation | Cancer Research UK |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 06/2021 |
End | 05/2024 |
Description | Research Grant |
Amount | £204,308 (GBP) |
Organisation | CBD Solutions |
Sector | Private |
Country | United States |
Start | 06/2018 |
End | 12/2019 |
Description | Senior Research Fellowship |
Amount | £420,000 (GBP) |
Funding ID | ARUK-SRF2016B-2 |
Organisation | Alzheimer's Research UK |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 03/2017 |
End | 03/2022 |
Description | Small molecule inhibition of ATG4B for therapeutic strategies in pancreatic ductal adenocarcinoma |
Amount | £75,000 (GBP) |
Funding ID | 557595 |
Organisation | University College Hospital |
Sector | Hospitals |
Country | United Kingdom |
Start | 03/2021 |
End | 02/2022 |
Description | Synthetic lethality screen to determine tyrosine phosphatase signaling mechanisms in neuroblastoma cells |
Amount | £7,300 (GBP) |
Organisation | University College London |
Sector | Academic/University |
Country | United Kingdom |
Start | 03/2021 |
End | 02/2023 |
Description | TDRF |
Amount | £151,213 (GBP) |
Funding ID | BB/P027431/1 |
Organisation | Biotechnology and Biological Sciences Research Council (BBSRC) |
Sector | Public |
Country | United Kingdom |
Start | 01/2018 |
End | 02/2019 |
Description | Targeting MYC through next generation structure-function |
Amount | £200,000 (GBP) |
Funding ID | DRCPLT-Nov20100001 |
Organisation | University College London |
Sector | Academic/University |
Country | United Kingdom |
Start | 03/2021 |
End | 01/2023 |
Description | Targeting autophagy dependence in pancreatic cancer |
Amount | £109,341 (GBP) |
Funding ID | 2018RIF_15 |
Organisation | Pancreatic Cancer UK |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 08/2019 |
End | 09/2023 |
Description | Templated Conversion of Transmissible Proteins in Neuromuscular Disease |
Amount | $427,500 (USD) |
Funding ID | 1R21 NS101588-01 |
Organisation | National Institutes of Health (NIH) |
Sector | Public |
Country | United States |
Start | 05/2018 |
End | 06/2021 |
Description | UCL Eisai collaboration |
Amount | £5,000,000 (GBP) |
Organisation | Eisai Ltd |
Sector | Private |
Country | Japan |
Start | 11/2017 |
End | 04/2019 |
Description | UCL RCIF Capital Equipment Fund 2018-19 |
Amount | £155,000 (GBP) |
Organisation | University College London |
Sector | Academic/University |
Country | United Kingdom |
Start | 08/2018 |
Description | UCL-Eisai Therapeutic Innovation Group: - Eisai Ltd: - UCL-Eisai Therapeutic Innovation Group (£ 60000; 2018 - 2019) |
Amount | £60,000 (GBP) |
Funding ID | 522585 |
Organisation | Eisai Ltd |
Sector | Private |
Country | Japan |
Start | 07/2018 |
End | 09/2019 |
Description | University of Pennsylvania Orphan Disease Center |
Amount | $51,020 (USD) |
Funding ID | MDBR-19-102-BPAN |
Organisation | University of Pennsylvania |
Sector | Academic/University |
Country | United States |
Start | 02/2019 |
End | 01/2020 |
Description | WT Institutional Strategic Support Fund |
Amount | £65,000 (GBP) |
Organisation | Wellcome Trust |
Sector | Charity/Non Profit |
Country | United Kingdom |
Start | 11/2015 |
End | 12/2017 |
Title | AAV Vectors for CNS expression |
Description | Investigation of the therapeutic potential of reduction of tau protein in CNS using truncated long non-coding RNA linked to the tau gene. Derivation of AAV9 vectors for CNS delivery of the long non-coding RNAs |
Type Of Material | Model of mechanisms or symptoms - mammalian in vivo |
Year Produced | 2018 |
Provided To Others? | No |
Impact | Project in progress. Current outcome: Wide CNS distribution after AAV9 injection in mouse model with reduction in brain levels of tau. We expect accompanying therapeutic effect. |
Title | Cellular FRET biosensor for tau aggregation |
Description | This is a cellular model with co-expression of GFP- and RFP-tau and is a robust model to study tau aggregation and assess the therapeutic potential of anti-aggregation agents and antibodies |
Type Of Material | Model of mechanisms or symptoms - in vitro |
Year Produced | 2018 |
Provided To Others? | No |
Impact | With this robust biosensor, we have shown the potential therapeutic anti-aggregation potential of anti-tau antibodies that we have developed. |
Title | Opera Phenix and Harmony Software |
Description | Application of the Opera Phenix and Harmony Analysis software for the automated, unbiased quantification of AAV transduction in complex mouse tissues |
Type Of Material | Technology assay or reagent |
Year Produced | 2020 |
Provided To Others? | No |
Impact | A paper describing this work has just been submitted for publication and will be place on Bioarchive if possible. |
Title | Stable cell lines for tau gene associated non-coding RNA genes |
Description | Stable overexpression of non-coding RNA genes enables consistent cell-based model to investigate the downstream regulatory effects of these non-coding genes |
Type Of Material | Model of mechanisms or symptoms - in vitro |
Year Produced | 2015 |
Provided To Others? | No |
Impact | Have succeeded in clarifying the precise role of the non-coding RNA genes in the post-transcriptional regulation of tau gene expression |
Title | Vimentin bundling assay (Opera Phenix) |
Description | High-throughput compound screening assay incorporating immunofluorescent analysis of vimentin intermediate filament structure and Opera Phenix confocal imaging to assess the efficacy of compounds on correcting the condensed vimentin bundling phenotype characteristic in ARSACS patient cells. |
Type Of Material | Technology assay or reagent |
Year Produced | 2020 |
Provided To Others? | No |
Impact | Potential to screen compounds for therapeutic alleviation of pathogenic vimentin bundiling in ARSACS patient cells. |
Description | ARUK DDI |
Organisation | University College London |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | The UKDP is working closely with the ARUK Drug Discovery Institute with the aim to develop novel therapies for neurodegenerative disorders. Both networks collaborate closely in terms of drug discovery research and through exchange of knowledge, equipment and technologies. |
Collaborator Contribution | The UKDP is working closely with the ARUK Drug Discovery Institute with the aim to develop novel therapies for neurodegenerative disorders. Both networks collaborate closely in terms of drug discovery research and through exchange of knowledge, equipment and technologies. |
Impact | None yet |
Start Year | 2017 |
Description | ASAP Consortium |
Organisation | Aligning Sciences Across Parkinson's |
Country | United States |
Sector | Charity/Non Profit |
PI Contribution | Provision of assays and expertise |
Collaborator Contribution | Shared Postdoc |
Impact | Collaboration network |
Start Year | 2021 |
Description | Autophagy |
Organisation | University College London |
Department | MRC Laboratory for Molecular Cell Biology |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | The collaboration is looking at abnormalities in monocyte autophagy in patients with SLE and APS. The post-doctoral researcher doing the work, Dr Vera Ripoll-Nunez, extracts monocytes from blood of patients and healthy controls and carries out all the experiments. The work was done using the Opera Phenix facility funded by MRC DPUK funding MR/M02492X/1 |
Collaborator Contribution | Professor Robin Ketteler and Dr Janos Kriston-Vizi in the Laboratory for Molecular and Cell Biology provide advice and expertise on the study of apoptosis as well as access to specialist confocal microscopy apparatus for measuring autophagic flux. |
Impact | We are developing a research project to look at the possibility of using modulators of autophagy as a novel therapeutic agent in lupus nephritis. We have been awarded a grant by the UCL Clinical Research and Development Committee to develop this project. |
Start Year | 2017 |
Description | CBD clinicopathological classification |
Organisation | University College London |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Part of team to investigate clinico-pathological correlates in progression of corticobasal degeneration and pathological seeding species. Participating with expert input into project and assisting in experimental procedures. |
Collaborator Contribution | They have described markers of clinico-pathological progression of tauopathy in corticobasal degeneration (CBD) and obtained funding for neuropathological classification of CBD sub-types and established methodology to investigate pathological seeding and spread in postmortem material. |
Impact | Ongoing. |
Start Year | 2017 |
Description | Dementia Platform UK Stem Cell Network |
Organisation | Cardiff University |
Department | School of Biosciences |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Developed a strategy for integrating stem cell neuronal research across 6 centres for harmonising and validating protocols, high content imaging of models, neurophysiology, genomics and proteomics, |
Collaborator Contribution | As above |
Impact | Two teaching technical workshops held at Oxford and Cambridge. |
Start Year | 2015 |
Description | Dementia Platform UK Stem Cell Network |
Organisation | University of Cambridge |
Department | Gurdon Institute |
Country | United Kingdom |
Sector | Charity/Non Profit |
PI Contribution | Developed a strategy for integrating stem cell neuronal research across 6 centres for harmonising and validating protocols, high content imaging of models, neurophysiology, genomics and proteomics, |
Collaborator Contribution | As above |
Impact | Two teaching technical workshops held at Oxford and Cambridge. |
Start Year | 2015 |
Description | Dementia Platform UK Stem Cell Network |
Organisation | University of Edinburgh |
Department | Centre for Clinical Brain Sciences (CCBS) |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Developed a strategy for integrating stem cell neuronal research across 6 centres for harmonising and validating protocols, high content imaging of models, neurophysiology, genomics and proteomics, |
Collaborator Contribution | As above |
Impact | Two teaching technical workshops held at Oxford and Cambridge. |
Start Year | 2015 |
Description | Dementia Platform UK Stem Cell Network |
Organisation | University of Manchester |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Developed a strategy for integrating stem cell neuronal research across 6 centres for harmonising and validating protocols, high content imaging of models, neurophysiology, genomics and proteomics, |
Collaborator Contribution | As above |
Impact | Two teaching technical workshops held at Oxford and Cambridge. |
Start Year | 2015 |
Description | Dementia Platform UK Stem Cell Network |
Organisation | University of Oxford |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Developed a strategy for integrating stem cell neuronal research across 6 centres for harmonising and validating protocols, high content imaging of models, neurophysiology, genomics and proteomics, |
Collaborator Contribution | As above |
Impact | Two teaching technical workshops held at Oxford and Cambridge. |
Start Year | 2015 |
Description | Drug Discovery Approaches for Mitophagy Modulators |
Organisation | Alzheimer's Research UK |
Department | UCL Drug Discovery Institute |
Country | United Kingdom |
Sector | Charity/Non Profit |
PI Contribution | We have had two joint grants to utilise a genetic siRNA and compound screen to find modulators of mitophagy. We have provided the original assays in low throughput and employed a research assistant based at the DDI to optimise in high throughput and perform the screens. |
Collaborator Contribution | The DDI provide expertise in screening, and the facilities and equipment necessary for high content screening (microscope, automated liquid handling etc. |
Impact | We received two grants from the UCL Transaltional research office - a therapeutic innovation fund, and a therapeutic acceleration support grant. |
Start Year | 2016 |
Description | Gamma secretase stabilisiers |
Organisation | Janssen Pharmaceutica NV |
Country | Belgium |
Sector | Private |
PI Contribution | Establishment of a high throughput screen to identify compounds that modulate gamma secretase enzyme activity. |
Collaborator Contribution | Supply of reagents (antibodies) and compound libraries |
Impact | The compound screen is currently being run. We hope to identify novel compounds to enable a drug discovery programme |
Start Year | 2018 |
Description | Industry collaboration - drug discovery in iPSC derived ALS models |
Organisation | Cerevance Ltd |
Country | United Kingdom |
Sector | Private |
PI Contribution | Based on a discovery made in our laboratory we proposed a platform utilising iPSC derived motor neurons to screen for modulators of early phenotypes. We provided the expertise, the platform and developed the screen in our laboratory. |
Collaborator Contribution | The partners provided resources for a postdoctoral scientist for 3 years, and also generated a gene edited control in their laboratory for use in the screen. |
Impact | This partnership resulted in a publication, and the outputs of the screen are currently being prepared for a further publication. |
Start Year | 2015 |
Description | Mitophagy |
Organisation | University College London |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Establishment of a phenotypic assay to enable identification of novel pathways for mitophagy in Parkinsons disease |
Collaborator Contribution | Helen Plun Favreau. Deep knowledge of Parkinsons disease and mitophagy biology. Follow up biological validation of hits identified in the siRNA screen. |
Impact | a publication is being prepared |
Start Year | 2017 |
Description | Modulation of astrocyte purinergic receptors to normalise neuronal hyper-excitability |
Organisation | Dementia Discovery Fund |
Country | United Kingdom |
Sector | Private |
PI Contribution | The identification and validation of novel purinergic receptor antagonists expressed by astrocytes. The demonstration that such antagonists are able to normalise the neuronal hyperexcitability in brain slices from mouse models of Alzheimer's disease. |
Collaborator Contribution | Funding and support to enable this project, and the establishment of a new company - AstronauTx |
Impact | None so far |
Start Year | 2019 |
Description | Modulation of astrocyte purinergic receptors to normalise neuronal hyper-excitability |
Organisation | University of Edinburgh |
Department | Edinburgh Neuroscience |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | The identification and validation of novel purinergic receptor antagonists expressed by astrocytes. The demonstration that such antagonists are able to normalise the neuronal hyperexcitability in brain slices from mouse models of Alzheimer's disease. |
Collaborator Contribution | Funding and support to enable this project, and the establishment of a new company - AstronauTx |
Impact | None so far |
Start Year | 2019 |
Description | NIH Rocky Mountain Lab |
Organisation | National Institute of Allergy and Infectious Diseases (NIAID) |
Department | Rocky Mountain Laboratories |
Country | United States |
Sector | Public |
PI Contribution | MRC: kinetic, mechanistic studies of small molecule inhibition of prion propagation in vitro |
Collaborator Contribution | RML: MRC: kinetic studies of small molecule inhibition of prion propagation from patient brain |
Impact | Publications: Brazilin Removes Toxic alpha-Synuclein and Seeding Competent Assemblies from Parkinson Brain by Altering Conformational Equilibrium, JMB 2021 conference abstracts and presentations: Biophysical Society 2020, 2019; KAUST 2019 |
Start Year | 2017 |
Description | Notum inhibitors |
Organisation | Francis Crick Institute |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Identification of small molecule inhibitors of the Wnt signaling inhibitor enzyme Notum |
Collaborator Contribution | Oxford - X ray crystal structure of Notum to enable the drug discovery Crick - generation of genetically modified mice for understanding of the functional rolel of notum |
Impact | Papers: Fish PV, Steadman, D, Bayle ED, Whiting PJ (2019). New Approaches for the Treatment of Alzheimer's Disease. Bioorg. Med. Chem Lett. 29: 125-131 Atkinson BN, Steadman D, Zhao, Sipthorp J, Vecchia L, Ruza RR, Jeganathan F, Lines G, Frew S, Monaghan A, Kjaer S, Bictash M, Jones EY, Fish PV. Discovery of 2-phenoxyacetamides as Inhibitors of the Wnt-depalmitoleating enzyme NOTUM from an X-ray Fragment Screen. Med. Chem. Comm. Submitted February 2019. |
Start Year | 2017 |
Description | Notum inhibitors |
Organisation | University of Oxford |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Identification of small molecule inhibitors of the Wnt signaling inhibitor enzyme Notum |
Collaborator Contribution | Oxford - X ray crystal structure of Notum to enable the drug discovery Crick - generation of genetically modified mice for understanding of the functional rolel of notum |
Impact | Papers: Fish PV, Steadman, D, Bayle ED, Whiting PJ (2019). New Approaches for the Treatment of Alzheimer's Disease. Bioorg. Med. Chem Lett. 29: 125-131 Atkinson BN, Steadman D, Zhao, Sipthorp J, Vecchia L, Ruza RR, Jeganathan F, Lines G, Frew S, Monaghan A, Kjaer S, Bictash M, Jones EY, Fish PV. Discovery of 2-phenoxyacetamides as Inhibitors of the Wnt-depalmitoleating enzyme NOTUM from an X-ray Fragment Screen. Med. Chem. Comm. Submitted February 2019. |
Start Year | 2017 |
Description | Open Innovation Collaboration with AstraZeneca UK and MRC-LMB Cambridge (still ongoing - huge delay because focus of AZ on COVID) |
Organisation | AstraZeneca |
Country | United Kingdom |
Sector | Private |
PI Contribution | Exchange of information (confidential) |
Collaborator Contribution | Exchange of information (confidential) |
Impact | Ongoing |
Start Year | 2019 |
Description | PERK - Stanford |
Organisation | Stanford University |
Department | Department of Neurology Stanford |
Country | United States |
Sector | Academic/University |
PI Contribution | Knowledge and material exchange |
Collaborator Contribution | Knowledge and material exchange |
Impact | None yet |
Start Year | 2020 |
Description | PerkinElmer |
Organisation | Perkin Elmer |
Country | United States |
Sector | Private |
PI Contribution | Closely working together on the development of high-content screening technologies. We provide expertise and knowledge. |
Collaborator Contribution | We have received free hardware and software upgrades, e.g. free 5x objective and software upgrades. |
Impact | This has enabled the use of additional technologies on the platform, e.g. Presiscan, FRET. |
Start Year | 2017 |
Description | Proteomics analyses to detect proteins associated with lncRNA |
Organisation | University College London |
Department | Structural Molecular Biology |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | We provide the ChIRP samples, including lncRNA with associated proteins, ready for mass spectrometry analyses. We then carry out a full data analysis and mining based on the initially processed proteomics data we get back from our collaborators. |
Collaborator Contribution | Our partner uses these samples to carry out high-sensitivity mass spectrometry analyses, including initial QC and data analyses. |
Impact | na |
Start Year | 2018 |
Description | RNA dysregulation in ALS |
Organisation | University College London |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Myself: I am a Consultant Neurologist at the National Hospital for Neurology and Neurosurgery, a top 3 hospital worldwide for neurological treatment. My clinical expertise is in ALS diagnosis and treatment, and co-directs clinical service. My scientific research focuses on developing human iPSC models for ALS that display reliable pathogenic phenotypes; our laboratory is one of the few internationally that produces robust cultures of both motor neurons and astrocytes. We have deeply characterised our cultures Our research is co-located at the Francis Crick Institute, UK's flagship center for basic biomedical research. Crick provides advanced core facilities ranging from scientific computing and sequencing to gene-editing and high-throughput screening. We have an outstanding track-record of collaboration: Ule and Patani have also collaborated for >10 years, exploring the impact of RNA-processing in neural development and ALS. In 2017, the three of us published a study characterizing robust phenotypes in ALS motor neurons and astrocytes (Hall et al Cell Reports 2017); this directly led to our current collaborative discovery of aberrant intron retention in ALS (Luisier et al Nature Communications in 2018 and later recognised by the Paulo Gontigo International Prize in Medicine 2018). I provide the ALS disease platform, including patient-derived iPSC, post-mortem samples, and mouse models. As an active neurologist, I will ultimately take molecular findings to the clinic, accessing consented patient material; he has industry partnerships (including Takeda, Cerevance and Ono), so we are poised to translate mechanistic insights from this project into drug discovery. |
Collaborator Contribution | Jernej Ule is Professor of Molecular Neurobiology; he pioneered CLIP techniques and has applied them to study RNAs bound by ALS-associated proteins, including TDP43 and FUS. Ule provides the expertise for the molecular work, splicing perturbations, CLIP and proteomic experiments. Nicholas Luscombe is Professor of Computational Biology studying transcriptional and post-transcriptional regulation and their use in controlling biological processes such as development; he performs detailed statistical analyses of large-scale datasets to decode the information contained in genome sequences. Luscombe performs all the computational analysis to build statistical models, integrating both primary and publicly available genomic datasets, as well as imaging data from the cellular experiments. Study designs arise from continuous discussions between us; colocation means that fresh insights are iteratively cycled between discovery, interpretation and planning with minimal delay. |
Impact | Grants MRC Senior Clinical Fellowship awarded in 2018 Prizes 03/19 International 3Rs Prize for 2018 09/18 International Paulo Gontijo Prize in Medicine for 2018 06/16 Faculty of Brain Sciences (UCL) Excellence Award in Scientific Communication Publications: 1. Smethurst P, Risse E, Tyzack G, Mitchell JS, Taha DM, Chen Y, Newcombe J, Collinge, Sidle K*, Patani R*. Distinct responses of neurons and astrocytes to TDP-43 proteinopathy in amyotrophic lateral sclerosis. Brain. In Press (Corresponding author). 2. Pandya VA, Patani R*. Decoding the relationship between ageing and amyotrophic lateral sclerosis: a cellular perspective. Brain. 2019 Dec 18. pii: awz360. doi: 10.1093/brain/awz360. (Corresponding author). 3. Thelin EP, Hall CE, Tyzack GE, Frostell A, Giorgi-Coll S, Alam A, Carpenter KLH, Mitchell J, Tajsic T, Hutchinson PJ, Patani R*, Helmy A*. Delineating Astrocytic Cytokine Responses in a Human Stem Cell Model of Neural Trauma. J Neurotrauma. 2019 Sep 18. doi: 10.1089/neu.2019.6480. (Corresponding author). 4. Tyzack GE, Luisier R, Taha DM, Neeves J, Modic M, Mitchell JS, Meyer I, Greensmith L, Newcombe J, Ule J, Luscombe NM*, Patani R*. Widespread FUS mislocalization is a molecular hallmark of amyotrophic lateral sclerosis. Brain. 2019 Sep 1;142(9):2572-2580. doi: 10.1093/brain/awz217. (Corresponding author). 5. Malik B, Devine H, Patani R, La Spada AR, Hanna MG, Greensmith L. Gene expression analysis reveals early dysregulation of disease pathways and links Chmp7 to pathogenesis of spinal and bulbar muscular atrophy. Sci Rep. 2019 Mar 5;9(1):3539. doi: 10.1038/s41598-019-40118-3. 5. Ziff OJ, Patani R*. Harnessing cellular aging in human stem cell models of amyotrophic lateral sclerosis. Aging Cell. 2018 e12862. 6. Luisier R, Tyzack GE, Hall CE, Mitchell JS, Devine H, Taha DM, Malik B, Meyer I, Greensmith L, Newcombe J, Ule J, Luscombe NM*, Patani R*. Intron retention and nuclear loss of SFPQ are molecular hallmarks of ALS. Nature Communications. 2018 May 22;9(1):2010. (Corresponding author). 7. Peskett TR, Rau F, O'Driscoll J, Patani R, Lowe R, Saibil HR. A liquid to solid phase transition underlying pathological huntingtin exon1 aggregation. Molecular Cell. 2018 May 17;70(4):588-601.e6. 8. Maffioletti SM, Sarcar S, Henderson ABH, Mannhardt I, Pinton L, Moyle LA, Steele-Stallard H, Cappellari O, Wells KE, Ferrari G, Mitchell JS, Tyzack GE, Kotiadis VN, Khedr M, Ragazzi M, Wang W, Duchen MR, Patani R, Zammit PS, Wells DJ, Eschenhagen T, Tedesco FS.Three-Dimensional Human iPSC-Derived Artificial Skeletal Muscles Model Muscular Dystrophies and Enable Multilineage Tissue Engineering. Cell Reports. 2018 Apr 17;23(3):899-908. 9. Kelley KW, Ben Haim L, Schirmer L, Tyzack GE, Tolman M, Miller JG, Tsai HH, Chang SM, Molofsky AV, Yang Y, Patani R, Lakatos A, Ullian EM, Rowitch DH.Kir4.1-Dependent Astrocyte-Fast Motor Neuron Interactions Are Required for Peak Strength. Neuron. 2018 Apr 18;98(2):306-319.e7. 10. Serio A, Patani R*. Concise Review: The Cellular Conspiracy of Amyotrophic Lateral Sclerosis. Stem Cells. 2018 Mar;36(3):293-303. 11. R. Simone, Balendra R, Moens TG, Preza E, Wilson KM, Heslegrave A, Jaramillo JG, Abdelkarim S, Clarke M, Woodling NS.... Patani R*, Fratta P*, Isaacs AM*. 'G-quadruplex- binding small molecules ameliorate C9orf72 FTD/ALS pathology in vitro and in vivo'. EMBO Molecular Medicine. 2018 Jan;10(1):22-31. (joint senior and corresponding author). 12. Tyzack GE, Hall CE, Sibley CR, Cymes T, Forostyak S, Carlino G, Meyer I, Schiavo G, Zhang SC, Gibbons GM, Newcombe J, Patani R*, Lakatos A* 'EphB1 is a neuronal signal that induces a neuroprotective astrocyte state, but fails in ALS' Nature Communications 2017 Oct 27;8(1):1164. (*Joint senior and corresponding author). 13. Thelin EP, Hall CE, Gupta K, Carpenter KLH, Chandran S, Hutchinson PJ, Patani R*, Helmy A*. Elucidating pro-inflammatory cytokine responses following traumatic brain injury in a human stem cell model.Journal of Neurotrauma 2017 Oct 5. doi: 10.1089/neu.2017.5155. (*Joint senior and corresponding author). 14. Hall CE, Yao Z, Choi M, Tyzack GE, Serio A, Luisier R, Harley J, Preza E, Arber C, Crisp SJ, Watson PMD, Kullmann DM, Abramov AY, Wray S, Burley R, Loh SHY, Martins LM, Stevens MM, Luscombe NM, Sibley C, Lakatos A, Ule J, Gandhi S*, Patani R*. 'Progressive motor neuron pathology and the role of astrocytes in a human stem cell model of VCP-related ALS'. Cell Reports 2017 May 30;19(9):1739-1749. (Corresponding author). 15. Soreq L, UK Brain Expression Consortium, North American Brain Expression Consortium, Rose J, Soreq E, Hardy J, Trabzuni D, Cookson MR, Smith C, Ryten M, Patani R*, Ule J*. 'Major shifts in glial regional identity are a transcriptional hallmark of human brain aging'. Cell Reports 2017 Jan 10;18(2):557-570. (*Joint senior and corresponding author). 12. Devine H, Patani R. 'The Translational Potential of Human Induced Pluripotent Stem Cells for Clinical Neurology'. Cell Biology and Toxicology 2017 Apr;33(2):129-144. 16. Smethurst P, Newcombe J, Troakes C, Simone R, Chen YR, Patani R, Sidle K. 'In vitro prion-like behaviour of TDP-43 in ALS'. Neurobiol Dis. 2016 S0969-9961(16)30195-4. 17. Tyzack G, Lakatos A, Patani R. 'Human Stem Cell-Derived Astrocytes: Specification and Relevance for Neurological Disorders'. Current Stem Cell Reports 2016 2:236-247. 18. Zirra A, Wiethoff S, Patani R. 'Neural conversion and patterning of human pluripotent stem cells: a developmental perspective'. Stem Cells International 2016 8291260. doi: 10.1155/2016/8291260. 19. Balendra R, Patani R. 'Quo vadis MND?'. World J Methodol. 2016 26;6(1):56-64. 20. Patani R. Generating Diverse Spinal Motor Neuron Subtypes from Human Pluripotent Stem Cells. Stem Cells International 2016 1036974. doi: 10.1155/2016/1036974. 21. Wiethoff S, Arber C, Li A, Wray S, Houlden H, Patani R. Using human induced pluripotent stem cells to model cerebellar disease: Hope and hype. J Neurogenet. 2015 Jun-Sep;29(2- 3):95-102. This work emphasises the importance of interdisciplinary and team science, integrating stem cell biology, developmental biology, neuropathology, bioinformatics and RNA biology. |
Start Year | 2018 |
Description | Radiation protection |
Organisation | University College London |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | I have a collaboration with Prof. Maria Hawkins. This is extremely productive collaboration where we complement each other's work, with the final aim to translate our findings to help patients. |
Collaborator Contribution | My partner is providing extensive clinical knowledge and in-depth radiation expertise. |
Impact | This is a multi-disciplinary collaboration involving biology, physics, mathematics. |
Start Year | 2020 |
Description | Simon-Raj In vivo work |
Organisation | University College London |
Department | Institute for Women's Health |
Country | United Kingdom |
Sector | Academic/University |
PI Contribution | Indentification and characterisation of tau gene promoter associated long-non-coding RNA gene involved in regulation of tau gene. Design of active minimised variants and derivation of AAV expression vectors for in vivo work |
Collaborator Contribution | Maintenance of mouse colony, AAV injection and behavioural studies |
Impact | Ongoing. One publication has resulted from collaboration (PMID: 30081233) |
Start Year | 2016 |
Description | Targeting ATG4B in cancer |
Organisation | AstraZeneca |
Country | United Kingdom |
Sector | Private |
PI Contribution | We are working closely together with AstraZeneca to identify small molecule inhibitors of autophagy. We have received a small molecule library from AstraZeneca and will perform the high-throughput screening in our lab. Validation of hits will be performed jointly in a collaboration. |
Collaborator Contribution | Provision of small molecule compounds and expertise/advice. |
Impact | Provision of small molecule compound library and expertise in drug discovery |
Start Year | 2020 |
Description | Tau-based gene therapy for neurodegenerative disorders |
Organisation | Karolinska University Hospital |
Department | Center for Molecular Medicine |
Country | Sweden |
Sector | Hospitals |
PI Contribution | We have developed vectors to test the concept of gene therapy directed at tau gene expression in slowing or halting the progress of tau-relaed neurodegeneration. |
Collaborator Contribution | Injection and study of mouse models for tauopathy |
Impact | None to date - still in progress |
Start Year | 2015 |
Description | Testing ATG4B inhibitors in cell-based assays |
Organisation | University of Antwerp |
Country | Belgium |
Sector | Academic/University |
PI Contribution | The partner provides us with small molecule compounds targeting the autophagy protease ATG4B for testing in cell-based assays that we have developed. |
Collaborator Contribution | The partner provides us with small molecule compounds targeting the autophagy protease ATG4B for testing in cell-based assays that we have developed. |
Impact | Research data that will be published eventually. |
Start Year | 2020 |
Title | ANTI-TAU ANTIBODIES AND USES THEREOF |
Description | Provided herein are antibodies that specifically bind Tau and methods of using the same. |
IP Reference | WO2019077500 |
Protection | Patent application published |
Year Protection Granted | 2019 |
Licensed | Commercial In Confidence |
Impact | E2814 antibody developed with TIG collaboration between UCL and Eisai. Pre-clinical studies leading to first-in-man trials (Phase 1) in Alzheimer's disease initiated in early 2020. |
Title | MEANS FOR MODULATING GENE EXPRESSION |
Description | The use of coding-gene linked long-non-coding RNA gene transcripts for transcriptional regulation of gene expression - as a therapeutic or research tool. |
IP Reference | GB1608907.0 |
Protection | Patent application published |
Year Protection Granted | 2016 |
Licensed | No |
Impact | Collaborations to test concept in vivo and approach by potential investors to for spin out company. |
Title | E2814 anti-tau Clinical Trial |
Description | Therapeutic intervention Phase I clinical trials for Alzheimer's disease to commence in April 2019 |
Type | Therapeutic Intervention - Drug |
Current Stage Of Development | Early clinical assessment |
Year Development Stage Completed | 2019 |
Development Status | Under active development/distribution |
Impact | Still under assessment |
Title | lncRNAs and therapeutic reduction of tau |
Description | Refined/truncated lncRNA transcript delivery in vivo by AAV vectors to test if tau is reduced and if there is any therapeutic benefits in animal models of tauopathies. Most recent funding from Brain Research Trust. |
Type | Therapeutic Intervention - Cellular and gene therapies |
Current Stage Of Development | Refinement. Non-clinical |
Year Development Stage Completed | 2016 |
Development Status | Under active development/distribution |
Impact | Possibly using the same concept to regulate translation of other genes |
Company Name | AstronauTx |
Description | AstronauTx develops pharmaceuticals for the treatment of neurodegenerative diseases such as Alzheimer's. |
Year Established | 2019 |
Impact | None as yet |
Website | https://astronautx.bio/ |
Company Name | Virology Research Services |
Description | Virology Research Services provides a range of outsourced virology testing and analysis services. |
Year Established | 2018 |
Impact | Screening anti-viral compounds |
Website | http://virologyresearchservices.com |
Description | @PataniLab Twitter |
Form Of Engagement Activity | Engagement focused website, blog or social media channel |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | We have over 1300 followers on Twitter and regularly tweet about updates from our lab |
Year(s) Of Engagement Activity | 2015,2016,2017,2018,2019,2020 |
URL | https://twitter.com/PataniLab |
Description | Conference: Glia in Health and Disease, Cold Spring Harbor Meeting July 16-19th 2020 |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | International conference on Glia and Health and Disease |
Year(s) Of Engagement Activity | 2020 |
Description | FCS single molecule workshop in Hyderabad |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Postgraduate students |
Results and Impact | About 300 students from India and from other countries attended this workshop and scientific conference, many of whom came from structurally disadvantaged backgrounds. This workshop introduces postgraduate and undergraduate students to single molecule spectroscopy techniques both in theory and in practice. In addition to lectures and seminars, the students got the unique opportunity to build their own microscopy setups and have extensive hands-on tutoring on super-resolution microscopy, Raman spectroscopy, and single molecule dynamics. Protein folding and misfolding processes in diseases like Alzheimer and prion disease were a central application of these techniques. In the conference following the workshop, the students had the opportunity to present their own research and discuss with leaders in the fields of single molecule spectroscopy and protein misfolding. The event was also attended by international companies and Indian science policy makers. |
Year(s) Of Engagement Activity | 2019 |
URL | https://fcs2019.tifrh.res.in/ |
Description | High-Content Biology Lab Opening Symposium |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Other audiences |
Results and Impact | 120 researchers attended the Opening Symposium for the High-Content Screening facility, which sparked interest in accessing the facility from academics and industry partners. |
Year(s) Of Engagement Activity | 2018 |
Description | Lab Tour for Pancreatic Cancer UK and General Public |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Supporters |
Results and Impact | This event was a Lab Tour, sponsored by Pancreatic Cancer UK, for supporters, patients and carers of patients with Pancreatic Cancer. I have presented my lab/facility in a talk and a lab tour, which sparked a lively discussion with the attendees. |
Year(s) Of Engagement Activity | 2019 |
Description | MRC DPUK Stem Cell Partnership workshop |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Postgraduate students |
Results and Impact | 2 day workshop for postdocs, PhD students and PIs on biology of neurodegenerative disease biology and use of stem cell derived models to study these. Researchers/students from 10-15 HEI attended |
Year(s) Of Engagement Activity | 2018 |
Description | Panel discussion at Royal Institution |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | National |
Primary Audience | Public/other audiences |
Results and Impact | Making monsters? Ethical gene editing Modern stem cell and embryology research has the potential to revolutionise medicine, but some see it as 'playing God'. I was part of a panel of scientists and policy experts discussing the latest developments in genetic medicine and whether we are ready for the ethical challenges these technologies raise. |
Year(s) Of Engagement Activity | 2018 |
URL | https://www.rigb.org/whats-on/events-2018/april/public-making-monsters-ethical-gene-editing |
Description | Profile piece by Nature Medicine |
Form Of Engagement Activity | A magazine, newsletter or online publication |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Professional Practitioners |
Results and Impact | Nature Medicine, 2020: 'Human stem cell models of disease and the prognosis of academic medicine' by Patani R. 2020 Apr;26(4):449. doi: 10.1038/s41591-020-0814-7. https://doi.org/10.1038/s41591-020-0814-7 |
Year(s) Of Engagement Activity | 2020 |
URL | https://doi.org/10.1038/s41591-020-0814-7 |
Description | Royal visit by HRH Princess Anne |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Policymakers/politicians |
Results and Impact | Visit from HRH Princess Anne to the High-Content Biology Screening laboratory, as part of the opening of the UCL-Zeiss Multimodal Imaging Centre. |
Year(s) Of Engagement Activity | 2020 |
Description | School Visit |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | About 20 A-level school children visited the High-Content Biology Laboratory and were given an introduction to lab equipment and work practices. The school reported increased interested in STEM based subjects from their pupils following the visit. |
Year(s) Of Engagement Activity | 2020 |
Description | School visit |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Local |
Primary Audience | Schools |
Results and Impact | Approx 20 school children visited the screening lab and were given hands-on introduction to research in my lab. |
Year(s) Of Engagement Activity | 2019 |
Description | Scientific Advisory Board Expert Panel for the Dementia Discovery Fund |
Form Of Engagement Activity | A formal working group, expert panel or dialogue |
Part Of Official Scheme? | No |
Geographic Reach | International |
Primary Audience | Industry/Business |
Results and Impact | The Scientific Advisory Board of the Dementia Discovery Fund in collaboration with Alzheimers Research UK, invited me as an expert panel member to discuss the role of Autophagy in Alzheimer's disease. The SAB (composed of leading experts in the pharmaceutical industry and charities) explored the possbility to invest in the development of autophagy modulating drugs for treatment of Alzheimer's disease. This discussion had an immediate impact on funding decisions and the policy of the Dementia Discovery Fund going forward in this area. |
Year(s) Of Engagement Activity | 2019 |
Description | Single cell imaging |
Form Of Engagement Activity | Participation in an activity, workshop or similar |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Schools |
Results and Impact | Have for the last three years had a group of year 12 students from Brighton College visit my lab and be introduced to the work we do on Dementia. We give a talk and then they visit different parts of the lab where my lab members tell they what experiments they do and what equipment we use. We also give out leaflets from the ARUK and ASoc. We discuss careers in neuroscience and especially dementia research. I have also had individual students undertake work experience in my lab for 2-5 days where we teach them about dementia and how we do experiments. They are introduced to the equipment and where and how we get funding to conduct our research. |
Year(s) Of Engagement Activity | 2016,2017,2018 |
Description | Talk on research as a career for girls |
Form Of Engagement Activity | A talk or presentation |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Schools |
Results and Impact | As a female in science, I gave a talk to school children at Leytonstone school on career as a researcher in neurodegeneration. |
Year(s) Of Engagement Activity | 2019 |
Description | Visit to high content imaging lab by HRH Princess Anne |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Supporters |
Results and Impact | Official Royal opening of imaging facilities at UCL by HRH Princess Anne. Royal Party toured labs and asked questions regarding the technology and research performed. |
Year(s) Of Engagement Activity | 2020 |
Description | visit by sixth form students to our labs |
Form Of Engagement Activity | Participation in an open day or visit at my research institution |
Part Of Official Scheme? | No |
Geographic Reach | Regional |
Primary Audience | Schools |
Results and Impact | 15 sixth form students from local state schools studying science with interest in medicine, neuroscience. Saw brain in brain bank, histology, stem cell derived neuronal cultures and questioned phd student, postdoc, medics and a patient with neurodegenerative disease. |
Year(s) Of Engagement Activity | 2018,2019 |