TY - JOUR
T1 - Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model
AU - Martino, S.
AU - Cavalieri, C.
AU - Emiliani, C.
AU - Dolcetta, D.
AU - De Cusella Angelis, M. G.
AU - Chigorno, V.
AU - Severini, G. M.
AU - Sandhoff, K.
AU - Bordignon, C.
AU - Sonnino, S.
AU - Orlacchio, A.
PY - 2002/8
Y1 - 2002/8
N2 - 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.
AB - 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.
KW - Bone marrow-derived stromal cells
KW - Cell therapy
KW - Hexosaminidase, Tay-Sachs
UR - http://www.scopus.com/inward/record.url?scp=0036694921&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0036694921&partnerID=8YFLogxK
U2 - 10.1023/A:1020256924099
DO - 10.1023/A:1020256924099
M3 - Article
C2 - 12374215
AN - SCOPUS:0036694921
VL - 27
SP - 793
EP - 800
JO - Neurochemical Research
JF - Neurochemical Research
SN - 0364-3190
IS - 7-8
ER -