GENE THERAPY OF TAY-SACHS
- 3 Anni 1999/2002
- 74.370€ Totale Fondi
Descrizione non disponibile
Pubblicazioni Scientifiche
- 2002 JOURNAL OF BIOLOGICAL CHEMISTRY
Absence of metabolic cross-correction in Tay-Sachs cells. Implications for gene therapy.
- 2005 HUMAN MOLECULAR GENETICS
A direct gene transfer strategy via brain internal capsule reverses the biochemical defect in Tay-Sachs disease
- 2002 NEUROCHEMICAL RESEARCH
Restoration of the GM2 ganglioside metabolism in bone marrow-derived stromal cells from Tay-Sachs disease animal model
