Liver-specific knockdown of M6PR with RNAi therapeutics to increase enzyme delivery to muscle tissues of Pompe disease mice during ERT

Administered By

Pediatrics, Medical Genetics

Awarded By

Alnylam Pharmaceuticals

Contributors

Kishnani, Priya Sunil Co Investigator
Sun, Baodong Principal Investigator

Start/End

February 9, 2018 - June 30, 2018