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UCL Institute of Ophthalmology

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Moosajee Lab

Led by Professor Mariya Moosajee.

We aim to understand the molecular basis of inherited eye disorders, in particular, the genetics of retinal dystrophies and ocular maldevelopment such as microphthalmia, anophthalmia, ocular coloboma and anterior segment dysgenesis. We are developing novel therapeutic approaches for genetic eye disease including nonsense suppression therapy, anti-apoptotic agents, and gene therapy using non-viral gene delivery systems including scaffold matrix attachment region (S/MAR) vectors. Proof-of-concept studies testing the safety and efficacy of novel small molecule drugs, such as ataluren and designer aminoglycosides, on (i) in vivo zebrafish models of genetic eye disease including ocular coloboma, microphthalmia, Choroideremia, Leber’s congenital amaurosis and Retinitis Pigmentosa; and (ii) in vitro patient cells including skin fibroblasts and induced pluripotent stem cell (iPSC) derived retinal pigment epithelium, photoreceptors and optic cups with the same ocular disease.