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First report of a mutant viral vector with potential to improve efficiency in treating severe LCA

A research team, based at the RC Byrd Health Sciences Centre, West Virginia University, has shown that a self complimentary AAV (adeno-associated virus) vector with a specific capsid alteration can increase the transduction efficiency of a replacement gene therapy for LCA (Leber congenital amaurosis). The research, conducted by Dr. Cristy Ku and Professor Visvanathan Ramamurthy, represented the first ever demonstration in a rapid retinal degeneration model using 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.