HIV Tat Domain Improves Cross-correction of Human Galactocerebrosidase in a Gene- and Flanking Sequence-dependent Manner

• Váldodahkkit: Xing-Li Meng (Dahkki), Yoshikatsu Eto (Dahkki), Raphael Schiffmann (Dahkki), Jin-Song Shen (Dahkki)
• Materiálatiipa: Girji
• Almmustuhtton: Elsevier, 2013-01-01T00:00:00Z.
• Fáttát: article
• Liŋkkat: Connect to this object online.

Krabbe disease is a devastating neurodegenerative lysosomal storage disorder caused by a deficiency of β-galactocerebrosidase (GALC). Gene therapy is a promising therapeutic approach for Krabbe disease. As the human brain is large and it is difficult to achieve global gene transduction, the efficacy...