870. Gene Transfer in the Sandhoff Murine Model Using Lentiviral Vectors

Sandhoff disease is an autosomal recessive neurodegenerative disease characterized by the intralysosomal accumulation of GM2 ganglioside. It is due to mutations in the hexosaminidases β-chain gene, resulting in a hexosaminidases A (αβ) and B (ββ) deficiency. This neurolipidosis, characterized by a predominant involvement of the central nervous system, has a dramatic evolution leading to early death. There is no efficient treatment for GM2 gangliosidosis to date.