Unlocking the inherent power of adeno-associated virus vectors for liver-targeted gene therapy

基于腺相关病毒（AAV）的基因工程病毒在肝脏和其他器官的遗传性疾病的治疗中显示出巨大的前景。目前，基于AAV的基因转移足以治疗诸如血友病B的病症，但大多数潜在的可治疗的肝脏病症是遥不可及的。这项资助的重点是改造AAV的外壳，以显着提高人类肝细胞基因修复的效率。

Genetically engineered viruses based on Adeno-Associated Virus (AAV) show immense promise in the treatment of genetic diseases of the liver and other organs. At present AAV-based gene transfer is sufficiently powerful to treat conditions such as haemophilia B, but the majority of potentially treatable conditions of the liver lie beyond reach. This grant focuses on engineering the outer shell of AAV to dramatically improve the efficiency with which human liver cells can be genetically repaired.