Available Technologies

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Search Results - samuel jacobson

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Gene therapy for the treatment of retinal degeneration and vision loss
Brief Description: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:Retinitis pigmentosa (RP) is a genetic form of early-onset retinal degenerati...
Published: 6/7/2018   |   Inventor(s): William Beltran, Gustavo Aguirre, Artur Cideciyan, Samuel Jacobson
Category(s): Therapeutics & Vaccines, Veterinary & Animal Health
Gene therapy for Leber congenital amaurosis ciliopathy
AAV-mediated gene therapy approach for the treatment of Leber congenital amaurosis retinopathies resulting from NPHP5 mutationsInventorDr. Gustavo Aguirre, Dr. William Beltran, Dr. Samuel Jacobson, Dr. Artur CideciyanProblemLeber congenital amaurosis (LCA) is an autosomal recessive disorder characterized by retinal dystrophy and severe visual impai...
Published: 9/21/2016   |   Inventor(s): Gustavo Aguirre, William Beltran, Samuel Jacobson, Artur Cideciyan
Category(s): Therapeutics & Vaccines