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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...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Nature Publishing Group
2013
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| 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 |