Bicistronic lentiviral vector corrects β-hexosaminidase deficiency in transduced and cross-corrected human Sandhoff fibroblasts

Bicistronic lentiviral vector corrects β-hexosaminidase deficiency in transduced and cross-corrected human Sandhoff fibroblasts

Sandhoff disease is an autosomal recessive neurodegenerative disease characterized by a GM2 ganglioside intralysosomal accumulation. It is due to mutations in the β-hexosaminidases β-chain gene, resulting in a β-hexosaminidases A (αβ) and B (ββ) deficiency. Mono and bicistronic lentiviral vectors co...

Full description

Bibliographic Details
Main Authors: Audrey Arfi, Christophe Bourgoin, Luisa Basso, Carla Emiliani, Brunella Tancini, Vanna Chigorno, Yu-Teh Li, Aldo Orlacchio, Livia Poenaru, Sandro Sonnino, Catherine Caillaud
Format: Article
Language:English
Published: Elsevier 2005-11-01
Series:Neurobiology of Disease
Subjects:
Gene therapy
Sandhoff disease
Lentiviral vector
Cross-correction
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996105001336

Internet

http://www.sciencedirect.com/science/article/pii/S0969996105001336

Similar Items