Innovative gene therapy for autosomal dominant retinitis pigmentosa

by DBGen Ocular Genomics

Mutations in the RHO gene are the main cause of dominant forms of retinitis pigmentosa (RP). RHO dictates the production of rhodopsin, a transmembrane protein present in large quantities in the outer ...

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Advances in gene therapy for retinitis pigmentosa due to RPGR gene mutation

by DBGen Ocular Genomics

In the first months of 2020, positive results in gene therapy of X-linked retinitis pigmentosa (XLRP) have been reported. XLRP is an inherited genetic disorder that causes progressive vision loss, mai...

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First patient treated in ProQR STELLAR phase 1/2 gene therapy clinical trial for Usher Syndrome Type II

by DBGen Ocular Genomics

Usher syndrome is a genetic disorder that presents recessive inheritance and is characterized by hearing loss and retinitis pigmentosa, traits that may be total or partial.

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