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

基于腺相关病毒（AAV）的基因工程病毒在治疗肝脏和其他器官的遗传性疾病方面显示出巨大的希望。目前，基于aav的基因转移足以治疗血友病B等疾病，但大多数潜在可治疗的肝脏疾病仍遥不可及。这笔拨款的重点是对AAV外壳进行工程改造，以显著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.