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Chinese Journal of Clinical Pharmacology and Therapeutics ›› 2002, Vol. 7 ›› Issue (2): 181-183.

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Advance in the genetherapy of Gaucher' s disease

WANG Jie, YIN Cai-Ling   

  1. Department of Human Genetics, Graduate School of Public Health, University of Pittsburgh, Pittsburgh, PA, 15261 ,USA
  • Received:2001-09-15 Revised:2001-10-08 Online:2002-04-26 Published:2020-11-27

Abstract: Gaucher disease is a commonly inherited, autosomtic recessive lysosomal storage disorder, due to the mutation of structure gene encoding beta-glucocerebrosidase (GC)resulted in the deficiency of the enzyme .Besides enzyme replacement, there is no good ways to treat it .This review briefly introduced the phenotypes, pathologic physiology and general therapy to the disease, strongly stress the genetherapy .From animals to human, from in vitro to in vivo, the review detaily descibe the rapid progress made in genetherapy of Gaucher disease .

Key words: Gaucher disease, glucocerebrosidase (GC), enzyme replacement therapy, genetherapy

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