Efficacy and safety of AAV RPGR gene therapy in X-linked retinitis pigmentosa: a systematic review and meta-analysis.

What was studied

This review looked at gene therapy for X-linked retinitis pigmentosa. It is a severe inherited eye disease. It is linked to changes in the RPGR gene. The therapy adds gene copies using an AAV vector. It aims to preserve photoreceptor cells. The search ran up to July 2026. It found 571 records and included 12 clinical reports. The authors pooled efficacy and safety data.

What they found

Retinal sensitivity improved in 25 of 33 people. Visual function improved in 28 of 52 people. Adverse events occurred in 42 of 90 people. Eye inflammation occurred in 36 of 81 people. Raised eye pressure occurred in 22 of 63 people. Higher vector doses were linked to more inflammation and serious eye events.

What it means for patients

The authors say the therapy shows clinically relevant signals of function across several outcomes. They say the eye safety profile looks monitorable. They say clear outcome definitions can guide later development.

Why it is not certain yet

The data came from 12 clinical reports. Some groups were linked across reports. The abstract gives no comparison with untreated people. Safety signals rose at higher doses. The authors call for standard outcome definitions and long-term follow-up in several countries.

Source

Efficacy and safety of AAV RPGR gene therapy in X-linked retinitis pigmentosa: a systematic review and meta-analysis.

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