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

Research output: Contribution to journalArticlepeer-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 languageEnglish
Pages (from-to)793-800
Number of pages8
JournalNeurochemical Research
Volume27
Issue number7-8
DOIs
Publication statusPublished - Aug 2002

Keywords

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

ASJC Scopus subject areas

  • Biochemistry
  • Neuroscience(all)

Fingerprint Dive into the research topics of 'Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model'. Together they form a unique fingerprint.

Cite this