Biology and potential strategies for the treatment of GM2 gangliosides

被引：20

作者：

Chavany, C ^{[1
]}

Jendoubi, M ^{[1
]}

机构：

[1] NIH, Genet & Mol Immunol Sect, Bethesda, MD 20892 USA

来源：

MOLECULAR MEDICINE TODAY | 1998年 / 4卷 / 04期

关键词：

D O I：

10.1016/S1357-4310(98)01227-1

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

The G(M2) gangliosidoses are a group of heritable neurodegenerative disorders caused by excessive accumulation of the ganglioside G(M2) owing to deficiency in beta-hexosaminidase activity. Tay-Sachs and Sandhoff diseases have similar clinical phenotypes resulting from a deficiency in human exhosaminidase alpha and beta subunits, respectively. The lack of treatment for G(M2) gangliosidoses stimulated interest in developing animal models to understand the molecular mechanisms underlying the various forms of this disease and to test new potential therapies. In this review, we discuss the molecular biology of G(M2) gangliosidoses and the different strategies that have been tested in animal models for the treatment of this genetic disorder, including gene transfer and cell engraftment of neural stem cells engineered to express the hexosaminidase isoenzymes.

引用

页码：158 / 165

页数：8

共 36 条

[31] Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase [J].