RNA gene therapy targeting recurrent mutations in USH2A may be a future promising treatment option for patients with retinitis pigmentosa. Phase 1/2 trials to evaluate the safety and tolerability of therapy were completed in 2021 and demonstrated improvement in best corrected visual acuity, static perimetry, and optical coherence tomography (OCT) retinal imaging; no serious adverse effects were reported. Phase 2/3 trials are planned to follow.
Early studies have suggested that stem cell–based therapies may be beneficial in IRD. However, there are no proven effective cell-based treatments for retinal diseases.
Learn more about inherited retinal diseases.
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Cite this: Donny W. Suh. Fast Five Quiz: Management of Inherited Retinal Diseases - Medscape - Apr 27, 2022.