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Technological advances in delivery vectors may accelerate treatments for early onset retinal degenerations or early onset retinal degener

New research, published in the journal of Human Molecular Genetics, has demonstrated that a self-complimentary AAV (adeno-associated virus) vector with a specific capsid alteration can increase the efficiency of a gene therapy for LCA (Leber congenital amaurosis). The research, conducted by Dr. Cristy Ku and Professor Visvanathan Ramamurthy at the RC Byrd Health Sciences Centre, West Virginia University, represented the first ever demonstration in a rapid retinal degeneration model of a self complimentary AAV with mutations of surface-exposed tyrosine residues on the viral capsid. If successful in larger studies, the technology may have significant implications for broadening the spectrum of patients treatable with ocular gene therapy tools.