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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Detalles Bibliográficos
Autores principales: Meng, Xing-Li, Eto, Yoshikatsu, Schiffmann, Raphael, Shen, Jin-Song
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2013
Materias:
Methods - Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4027426/
https://www.ncbi.nlm.nih.gov/pubmed/24150577
http://dx.doi.org/10.1038/mtna.2013.57

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4027426/
https://www.ncbi.nlm.nih.gov/pubmed/24150577
http://dx.doi.org/10.1038/mtna.2013.57

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