Systemic delivery of nucleic acids via the pulmonary route - a non-invasive method of delivering genes and ribonucleic acids

Lead Research Organisation: University College London
Department Name: School of Pharmacy

Abstract

Gene replacement therapy and gene silencing products have been approved for use in humans
1
and these are administered by intravenous or local injections . For chronic conditions it would be
preferable to administer nucleic acids by non-parenteral routes. We hypothesise that the pulmonary route of administration could provide a means of achieving systemic exposure to gene therapeutics (protein expression outside of the lung). The delivery of genes and siRNA to the lung epithelial cells has been reported2, 3, however we are aiming for systemic exposure following pulmonary delivery and will explore ways in which this may be achieved via the non-invasive pulmonary route. We will design non-viral vectors utilising new chemistries and constructs with the aim of overcoming the challenges associated with pulmonary delivery: lung deposition, the mucosal barrier, uptake by lung epithelial cells and transfer to pulmonary capillaries, stability following capillary uptake and translocation to target organs.

Publications

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Studentship Projects

Project Reference Relationship Related To Start End Student Name
EP/S023054/1 01/10/2019 31/03/2028
2327432 Studentship EP/S023054/1 23/09/2019 21/09/2023 Savvas Dimiou