Researchers based at the University of Pennsylvania, Philadelphia have demonstrated that the use of adeno-associated virus serotype 8 (AAV8) is likely to represent the optimal approach to using gene therapy targeted at photoreceptor cells in the retina. The AAV8 vector and a similar construct (AAV2) have been used widely as delivery systems to carry a variety of gene constructs into human cells while AAV2 has been used in Phase I and II clinical trials to treat Leber congenital amaurosis (LCA). As photoreceptor cells are the primary cell type affected in many inherited retinal degenerations the characterization of safe and efficient vectors will be key to the advance of successful gene therapies for a variety of retinal disorders.
Next generation viral delivery systems target photoreceptors for therapy
- by swdadmin