Investigating pseudotyped lentiviral vector for gene delivery to the lung.

Lead Research Organisation: University of Oxford
Department Name: RDM Radcliffe Department of Medicine

Abstract

Gene therapy has the potential to treat the underlying cause of many inherited or acquired diseases. Lentiviral vectors offer a way of introducing a gene (transgene) into cells such that it is integrated into the host cell genome. This integration provides the possibility of long term expression as lentiviruses are capable of transducing both dividing and non-dividing cells. In this project the aim is to assess ability of two different sets of pseudotypes to transduce the cells of the lung (F/HN from the Sendai virus and HA/NA from the Influenza virus). The project will compare the ability of these sets of molecules, when pseudotyped onto the same lentiviral vector, to transduce various cell types within the lung and compare their ability to transduce cells that express the transgene for a long period of time. Depending on initial results the vector components may be modified in order to optimise gene transfer to the lungs for a specific lung disease decided upon based on cell types being successfully transduced. A therapeutic expressing vector may then be generated to carry forward for evaluation prior to scale up and clinical trials.

Publications

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Studentship Projects

Project Reference Relationship Related To Start End Student Name
MR/N018168/1 01/10/2016 30/03/2021
1808161 Studentship MR/N018168/1 01/10/2016 21/01/2021 Rosie Munday
 
Description Emerging gene therapies for CF 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Professional Practitioners
Results and Impact Presentation to a group of Health professionals informing on Emerging Gene Therapies for CF, which sparked questions and discussion afterwards.
Year(s) Of Engagement Activity 2018
 
Description Gene Therapy: A Brave New World 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Public/other audiences
Results and Impact Purpose was to enlighten audience about Gene Therapy and its potential which resulted in discussion and personal donations from members of the public.
Year(s) Of Engagement Activity 2018
 
Description Meet the Expert - ESGCT Spring School 
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 Students had the opportunity to talk with Experts in their field
Year(s) Of Engagement Activity 2018
 
Description Meet the Experts- ESGCT Spring School 
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 Students had the opportunity to talk with experts in their field of science.
Year(s) Of Engagement Activity 2018
 
Description Poster Presentation at ASGCT: High levels of native eGFP detected following F/HN lentiviral vector transduction of a variety of lung Models. 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Poster presentation sharing unpublished work with international field of experts in gene therapy.
Year(s) Of Engagement Activity 2019
 
Description Poster Presentation at ASGCT: Lentiviral Vector Pseudotyped with Sendai Virus F and HN Proteins Uses Sialylated Glycan Receptors to Efficiently Target Human Airway Cells. 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Poster presentation sharing unpublished work with international field of experts in gene therapy.
Year(s) Of Engagement Activity 2020
 
Description Poster Presentation at BSGCT: A method for assessing the distribution of eGFP expressing cells in the mouse lung following lentiviral vector transduction. 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact A poster and minute long oral presentation to share unpublished work and discuss with experts from the gene therapy field.
Year(s) Of Engagement Activity 2018
 
Description Where next for Gene Therapy of Cystic Fibrosis lung disease at UCL GOSICH - BDRC Seminar 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact 30-40 Professional Scientists attended my presentation on where next for Gene Therapy of Cystic Fibrosis Lung Disease, which sparked questions and discussion afterwards
Year(s) Of Engagement Activity 2018