Human gene therapy for glaucoma using CRISPR-Cas9
Research type
Research Study
Full title
Human gene therapy for glaucoma using CRISPR-Cas9 and investigated with eye bank ciliary body tissue.
IRAS ID
192997
Contact name
Andrew D Dick
Contact email
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
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. Treatment using eye drops has been available for many years, but they are expensive, have side-effects, need lifelong use and often don’t reduce the pressure enough. Surgery is effective, but requires highly trained surgeons, is potentially high risk, has a relatively long recovery period and can fail over time. More effective treatments are needed, particularly for use in the developing world where these same limitations are prohibitive.
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 will explore 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 human ciliary bodies 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. At this stage, genes known to be critical to aqueous humour production will be targeted. In theory this approach as a treatment could allow lifelong reduction in eye pressure following a single injection.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
16/SW/0124
Date of REC Opinion
25 Apr 2016
REC opinion
Favourable Opinion