Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model

S. Martino; C. Cavalieri; C. Emiliani; D. Dolcetta; M. G. De Cusella Angelis; V. Chigorno; G. M. Severini; K. Sandhoff; C. Bordignon; S. Sonnino; A. Orlacchio

doi:10.1023/A:1020256924099

Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model

S. Martino, C. Cavalieri, C. Emiliani, D. Dolcetta, M. G. De Cusella Angelis, V. Chigorno, G. M. Severini, K. Sandhoff, C. Bordignon, S. Sonnino, A. Orlacchio

IRCCS pediatrico Burlo Garofolo

Research output: Contribution to journal › Article › peer-review

Abstract

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.

Original language	English
Pages (from-to)	793-800
Number of pages	8
Journal	Neurochemical Research
Volume	27
Issue number	7-8
DOIs	https://doi.org/10.1023/A:1020256924099
Publication status	Published - Aug 2002

Keywords

Bone marrow-derived stromal cells
Cell therapy
Hexosaminidase, Tay-Sachs

ASJC Scopus subject areas

Biochemistry
Neuroscience(all)

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Martino, S., Cavalieri, C., Emiliani, C., Dolcetta, D., De Cusella Angelis, M. G., Chigorno, V., Severini, G. M., Sandhoff, K., Bordignon, C., Sonnino, S., & Orlacchio, A. (2002). Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model. Neurochemical Research, 27(7-8), 793-800. https://doi.org/10.1023/A:1020256924099

Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model. / Martino, S.; Cavalieri, C.; Emiliani, C.; Dolcetta, D.; De Cusella Angelis, M. G.; Chigorno, V.; Severini, G. M.; Sandhoff, K.; Bordignon, C.; Sonnino, S.; Orlacchio, A.

In: Neurochemical Research, Vol. 27, No. 7-8, 08.2002, p. 793-800.

Research output: Contribution to journal › Article › peer-review

Martino, S, Cavalieri, C, Emiliani, C, Dolcetta, D, De Cusella Angelis, MG, Chigorno, V, Severini, GM, Sandhoff, K, Bordignon, C, Sonnino, S & Orlacchio, A 2002, 'Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model', Neurochemical Research, vol. 27, no. 7-8, pp. 793-800. https://doi.org/10.1023/A:1020256924099

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abstract = "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.",

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AU - De Cusella Angelis, M. G.

AU - Chigorno, V.

AU - Severini, G. M.

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