PR001 gene therapy increased GCase activity and improved Gaucher disease type 1 phenotypes in mouse models

Publication date: February 2021Source: Molecular Genetics and Metabolism, Volume 132, Issue 2Author(s): Patty Sheehan, Laura D. Heckman, Li Chin Wong, Zhaohui Yang, Hsuan-Ni Lin, Sid Kamalakaran, Swetha Garimalla, Jason Politi, Yong Dai, Franz Hefti, Asa Abeliovich
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research