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