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

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...

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Hlavní autoři:	Xing-Li Meng, Yoshikatsu Eto, Raphael Schiffmann, Jin-Song Shen
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2013-01-01
Edice:	Molecular Therapy: Nucleic Acids
Témata:	corss-correction galactocerebrosidase protein transduction domain
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2162253116301883
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