Ophthalmogenetics and Gene Therapy

Ophthalmogenetics and gene therapy are transforming the treatment of hereditary eye diseases by targeting the genetic causes of vision disorders. Conditions like retinitis pigmentosa, Leber congenital amaurosis, and Stargardt disease result from genetic mutations affecting retinal cells. Gene therapy introduces or modifies genetic material to correct these defects, restoring or preserving vision. Luxturna, the first FDA-approved gene therapy for inherited retinal disease, has shown promising results. Advances in CRISPR and RNA-based therapies offer hope for more effective treatments. As research progresses, ophthalmogenetics continues to pave the way for personalized medicine, improving outcomes for patients with genetic eye conditions.

 

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