Restoration of the GM2 Ganglioside Metabolism in Bone Marrow–Derived Stromal Cells from Tay-Sachs Disease Animal Model
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- 作者：Martino ; S. ; Cavalieri ; C. ; Emiliani ; C. ; Dolcetta ; D. ; Cusella De Angelis ; M. G. ; Chigorno ; V. ; Severini ; G. M. ; Sandhoff ; K. ; Bordignon ; C. ; Sonnino ; S. ; Orlacchio ; A.
- 关键词：Hexosaminidase ; Tay ; Sachs ; cell therapy ; bone marrow ; derived stromal cells
- 刊名：Neurochemical Research
- 出版年：2002
- 出版时间：2002
- 年：2002
- 卷：27
- 期：7/8
- 页码：793-800
- 全文大小：2.58 MB
文摘
The therapeutic potential of bone marrow–derived stromal cells for the therapy of Tay-Sachs disease is primarily related to the restoration of their own GM2 ganglioside storage. With this aim, we produced bone marrow–derived stromal cells from the adult Tay-Sachs animal model and transduced them with a retroviral vector encoding for the α-subunit of the lysosomal enzyme β-hexosaminidase A (E.C. 3.2.1.52). Our results demonstrate that transduced Tay-Sachs bone marrow–derived stromal cells have β-hexosaminidase A comparable to that of bone marrow-derived stromal cells from wild-type mice. Moreover, β-hexosaminidase A in transduced Tay-Sachs bone marrow-derived stromal cells was able to hydrolyze the GM2 ganglioside in a feeding experiment, thus demonstrating the correction of the altered phenotype.