shRNA Adeno-associated Virus Serotype 2, pH1-(VARS2-shRNA-Seq3)(CAT#: AAV-SI2385WQ)

This product is a VARS2-shRNA encoding AAV, which is based on AAV-2 serotype. The VARS2 encodes a mitochondrial aminoacyl-tRNA synthetase, which catalyzes the attachment of valine to tRNA(Val) for mitochondrial translation. Mutations in this gene cause combined oxidative phosphorylation deficiency-20, and are also associated with early-onset mitochondrial encephalopathies. The expression of VARS2-shRNA leads to target gene silencing and this product can be used in gene therapy research and development.