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Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?
[Image: see text] Introduction: Krabbe disease (KD) is an autosomal recessive disorder caused by mutations in the galactocerebrosidase (GALC) gene resulting in neuro-inflammation and defective myelination in the central and peripheral nervous systems. Most infantile patients present with clinical fe...
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| Publicado no: | Bioimpacts |
|---|---|
| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Tabriz University of Medical Sciences (TUOMS Publishing Group)
2021
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8022232/ https://ncbi.nlm.nih.gov/pubmed/33842284 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.34172/bi.2021.21 |
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