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Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease
[Image: see text] Introduction: Krabbe disease (KD) is an autosomal recessive lysosomal disorder caused by mutations in the galactocerebrosidase (GALC) gene. This results in defective myelination in the peripheral and central nervous systems due to low GALC activity. Treatment at this time is limite...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Tabriz University of Medical Sciences
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7186542/ https://www.ncbi.nlm.nih.gov/pubmed/32363154 http://dx.doi.org/10.34172/bi.2020.13 |