Search Results - samuel+jacobson

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Gene Therapy for Leber Congenital Amaurosis Ciliopathy
AAV-mediated gene therapy approach for the treatment of Leber congenital amaurosis retinopathies resulting from NPHP5 mutations Problem: Leber congenital amaurosis (LCA) is an autosomal recessive disorder characterized by retinal dystrophy and severe visual impairment within the first year of life. LCA occurrence, while rare (1/50,000 – 1/33,000...
Published: 7/2/2024   |   Inventor(s): Gustavo Aguirre, William Beltran, Samuel Jacobson, Artur Cideciyan
Keywords(s):  
Category(s): Technology Classifications > Therapeutics
Gene Therapy for the Treatment of Retinal Degeneration and Vision Loss
Recombinant adeno-associated virus (rAAV) expressing the gene for retinitis pigmentosa GTPAse regulation (RPGR) for the functional and structural rescue of photoreceptor cells of the eye as gene therapy for retinal degeneration and vision loss. Technology Overview: Retinitis pigmentosa (RP) is a genetic form of early-onset retinal degeneration that...
Published: 7/8/2024   |   Inventor(s): William Beltran, Gustavo Aguirre, Artur Cideciyan, Samuel Jacobson
Keywords(s): Gene Therapy
Category(s): Technology Classifications > Animal Health, Technology Classifications > Therapeutics