Advance in the genetherapy of Gaucher's disease / 中国临床药理学与治疗学

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.