Unlocking the inherent power of adeno-associated virus vectors for liver-targeted gene therapy [ 2019 - ]

Research Grant

Brief description 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.

Funding Amount $691,653.00

Funding Scheme Project Grants

Notes Standard Project Grant