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PR001 gene therapy increased GCase activity and improved Gaucher disease type 1 phenotypes in mouse models
PR001 gene therapy increased GCase activity and improved Gaucher disease type 1 phenotypes in mouse models
2021
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
Keywords:
Disease
Phenotype
Cancer research
Genetic enhancement
Medicine
Correction
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