gene therapy for glaucoma and ocular immune mechanisms
Research type
Research Study
Full title
human gene therapy for glaucoma, multimodal analysis of ocular immune mechanisms, and immune responses to gene therapy.
IRAS ID
271896
Contact name
Andrew D Dick
Contact email
Sponsor organisation
Research & Enterprise Development, University of Bristol
Duration of Study in the UK
3 years, 0 months, 3 days
Research summary
Inflammation inside the eye is called uveitis and causes sight loss without treatment. Our work through combining novel imaging techniques will allow us to better understand how inflammation develops through the complex multilayered structures of the eye by characterising the populations of immune cells within these tissue layers and the changes in these populations during states of tissue inflammation. This will allow us to target treatments more precisely and at the right time during the course of uveitic eye disease.
Glaucoma is the leading cause of irreversible visual loss worldwide and an estimated 11.2 million people will be completely blind from the disease by 2020. Clinical trials have shown that reducing the pressure in the eye can prevent loss of vision from the commonest forms of glaucoma. Gene therapy using engineered viruses called AAV, to re-programme cells of the eye has been shown to be safe in recent clinical trials. This project explores using the same viruses to infect cells of the ciliary body. This is the part of the eye responsible for continually producing aqueous humour - the fluid that maintains the pressure of the eye. Using eyes donated for research from the Bristol Eye Bank, the virus will be programmed to deliver components of a system called CRISPR. This can cause genes to be accurately disrupted to stop them from making their encoded proteins.
In theory this approach as a treatment could allow lifelong reduction in eye pressure following a single injection. Our uveitis work will also allow us to model and better understand inflammation in response to gene therapy. Currently this is one of the barriers to efficacy of gene therapy identified in previous clinical trials of viral vector gene therapy in humans.
REC name
London - London Bridge Research Ethics Committee
REC reference
20/LO/0336
Date of REC Opinion
5 Mar 2020
REC opinion
Favourable Opinion