Refining gene therapy for glaucoma using post-mortem human tissue
Research type
Research Study
Full title
Refining gene therapy for glaucoma using post-mortem human tissue
IRAS ID
329242
Contact name
Colin Chu
Contact email
Sponsor organisation
Research and Enterprise Division, University of Bristol
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
Glaucoma is the leading cause of irreversible blindness worldwide. It is generally caused by chronically raised pressure in the eye that can damage the optic nerve and if left untreated, results in permanent vision loss.
Current treatments include eye drops, laser treatment and surgery however there are limitations to all these treatment options. The ciliary body (CB) in the eye is the source of fluid production and the balance with how much enters and exits the eye affects the pressure.
We are building on our research investigating the use of gene editing technologies such as CRISPR-Cas9. Aquaporin 1 (Aqp1) has been identified as a promising gene target in the ciliary body. Our research shows when injected in the mouse eye, CRISPR-Cas9 disruption of Aqp1 leads to a reduction of ocular pressure.
A method of delivery is necessary to directly transport the treatments into the eye. Our transporter is a deactivated engineered virus called adeno-associated virus (AAV). Our group has been able to set up an in-house AAV manufacturing unit and are developing AAV to specifically target the ciliary body to be able to direct our therapy to Aqp1 genes in the ciliary body and not in other eye tissues would reduce off-target effects.
We now need to use donor human eye tissues to test the different AAV and gene targets in the most appropriate way.
In the future, an AAV gene therapy to target Aqp1 in the CB may provide an effective and long-lasting option to treat Glaucoma.
REC name
HSC REC B
REC reference
23/NI/0072
Date of REC Opinion
16 May 2023
REC opinion
Favourable Opinion