shRNA Adeno-associated Virus Serotype 2, pH1-(Tmem231-shRNA-Seq3)(CAT#: AAV-SI2674WQ)

This product is a Tmem231-shRNA encoding AAV, which is based on AAV-2 serotype. The Tmem231 gene encodes a transmembrane protein, which is a component of the B9 complex involved in the formation of the diffusion barrier between the cilia and plasma membrane. Mutations in this gene cause Joubert syndrome (JBTS). The expression of Tmem231-shRNA leads to target gene silencing and this product can be used in gene therapy research and development.