Children with hemophilia (CwH) are advised to take special precautions when engaging in physical activity due to the risk of bleeding from an injury. The obesity epidemic [...]

Adeno-associated virus (AAV) is widely used in gene therapy but has limited integration, reducing long-term transgene expression. We engineered an AAV-rDNA vector to enha [...]

Adeno-associated virus (AAV) based in vivo gene therapy has made great progress in recent years including an approved treatment (Glybera) in Europe and several [...]