Gene therapy in metachromatic leukodystrophy

被引：0
作者：
Sevin, C.
Cartier-Lacave, N.
Aubourg, P. [1 ]
机构：
[1] Hop St Vincent de Paul, Dept Pediat Neurol, F-75014 Paris, France
来源：
INTERNATIONAL JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS | 2009年 / 47卷
关键词：
arylsulfatase A; brain gene therapy; hematopoietic stem cell gene therapy; metachromatic leukodystrophy; sulfatides; A-DEFICIENT MICE; MOUSE MODEL; MARROW TRANSPLANTATION; HEMATOPOIETIC-CELLS; NERVOUS-SYSTEM; ANIMAL-MODEL; DISEASE; PROTEIN; STORAGE; DIFFERENTIATE;
D O I：
暂无
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by deficiency of the lysosomal enzyme arylsulfatase A. Deficiency of this enzyme results in intralysosomal storage of sphingolipid cerebroside 3-sulfates (sulfatides), which are abundant in myelin and neurons. A pathological hallmark of MLD is demyelination and neurodegeneration, causing various and Ultimately lethal neurological symptoms. This review discusses the potential therapeutic application of hematopoietic stem cell gene therapy and intracerebral gene trans l-er (brain gene therapy) in patients with MLD.
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页码：S128 / S131
页数：4
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