Literature DB >> 12374215

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

S Martino1, C Cavalieri, C Emiliani, D Dolcetta, M G Cusella De Angelis, V Chigorno, G M Severini, K Sandhoff, C Bordignon, S Sonnino, A Orlacchio.   

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 alpha-subunit of the lysosomal enzyme beta-hexosaminidase A (E.C. 3.2.1.52). Our results demonstrate that transduced Tay-Sachs bone marrow-derived stromal cells have beta-hexosaminidase A comparable to that of bone marrow-derived stromal cells from wild-type mice. Moreover, beta-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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Year:  2002        PMID： 12374215     DOI： 10.1023/a:1020256924099

Source DB:  PubMed          Journal:  Neurochem Res        ISSN： 0364-3190            Impact factor:   3.996


  41 in total

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Journal:  Proc Natl Acad Sci U S A       Date:  2002-02-19       Impact factor: 11.205

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5.  Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

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Journal:  Hum Mol Genet       Date:  1999-05       Impact factor: 6.150

7.  Retrovirus-mediated enzymatic correction of Tay-Sachs defect in transduced and non-transduced cells.

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Authors:  A Consiglio; A Quattrini; S Martino; J C Bensadoun; D Dolcetta; A Trojani; G Benaglia; S Marchesini; V Cestari; A Oliverio; C Bordignon; L Naldini
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Review 5.  New Approaches to Tay-Sachs Disease Therapy.

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Review 6.  Adipose Stem Cell Translational Applications: From Bench-to-Bedside.

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Review 7.  Insight into Mechanobiology: How Stem Cells Feel Mechanical Forces and Orchestrate Biological Functions.

Authors:  Chiara Argentati; Francesco Morena; Ilaria Tortorella; Martina Bazzucchi; Serena Porcellati; Carla Emiliani; Sabata Martino
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8.  Functionalized Silica Star-Shaped Nanoparticles and Human Mesenchymal Stem Cells: An In Vitro Model.

Authors:  Chiara Argentati; Francesco Morena; Chiara Fontana; Ilaria Tortorella; Carla Emiliani; Loredana Latterini; Giulia Zampini; Sabata Martino
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  8 in total

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