PR001 gene therapy increased GCase activity and improved neuronopathic Gaucher disease phenotypes

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Patty Sheehan, Laura D. Heckman, Tim Fenn, Li Chin Wong, Stuart Nelson, Swetha Garimalla, Jorge Haller, Jennifer Daily, Jason Politi, Yong Dai, Franz Hefti, Asa Abeliovich
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research