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Gene therapy PR001 increased GCase activity and improved neuronopathic Gaucher disease phenotypes in mouse models
Gene therapy PR001 increased GCase activity and improved neuronopathic Gaucher disease phenotypes in mouse models
2020
Laura D. Heckman
Patty Sheehan
Tim Fenn
Li Chin Wong
Stuart Nelson
Swetha Garimalla
Jorge Haller
Jennifer Daily
Jason Politi
Yong Dai
Franz Hefti
Asa Abeliovich
Keywords:
Phenotype
Genetics
Diabetes mellitus
Neuronopathic Gaucher Disease
Immunology
Genetic enhancement
Biology
Correction
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