shRNA Adeno-associated Virus Serotype 2, pU6-(Vps13a-shRNA-Seq1)(CAT#: AAV-SI2215WQ)

This product is a Vps13a-shRNA encoding AAV, which is based on AAV-2 serotype. The protein encoded by Vps13a gene may control steps in the cycling of proteins through the trans-Golgi network to endosomes, lysosomes and the plasma membrane. Mutations in this gene cause the autosomal recessive disorder, chorea-acanthocytosis. The expression of Vps13a-shRNA leads to target gene silencing and this product can be used in gene therapy research and development.