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Human Molecular Genetics, Vol 7, 831-838, Copyright © 1998 by Oxford University Press

ARTICLES

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

J Guidotti, S Akli, L Castelnau-Ptakhine, A Kahn and L Poenaru
Institut Cochin de Genetique Moleculaire (ICGM), U129 INSERM, Universite Rene Descartes-Paris V, CHU Cochin-Port Royal, Paris, France. guidotti@cochin.inserm.fr

Tay-Sachs disease is a severe neurodegenerative disorder due to mutations in the HEXA gene coding for the alpha-chain of the alpha-beta heterodimeric lysosomal enzyme beta-hexosaminidase A (HexA). Because no treatment is available for this disease, we have investigated the possibility of enzymatic correction of HexA-deficient cells by HEXA gene transfer. Human HEXA cDNA was subcloned into a retroviral plasmid generating to G.HEXA vector. The best Psi-CRIP producer clone of G.HEXA retroviral particles was isolated, and murine HexA-deficient fibroblasts derived from hexa -/- mice were transduced with the G.HEXA vector. Transduced cells overexpressed the alpha-chain, resulting in the synthesis of interspecific HexA (human alpha-chain/murine beta- chain) and in a total correction of HexA deficiency. The alpha-chain was secreted in the culture medium and taken up by HexA-deficient cells via mannose-6-phosphate receptor binding, allowing for the restoration of intracellular HexA activity in non-transduced cells.
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