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 underway.
Preliminary data from ongoing clinical trials have shown the safety and efficacy of stem cell–based regenerative therapies for retinal dystrophies. However, more studies are needed before these therapies can be integrated into clinical practice.
Learn more about inherited retinal diseases.
Medscape © 2023 WebMD, LLC
Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.
Cite this: Donny W. Suh, Raj K. Maturi. Fast Five Quiz: Management of Inherited Retinal Diseases - Medscape - Mar 31, 2023.