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Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy
Adrenoleukodystrophy (ALD) is caused by mutations within the X-linked ABCD1 gene, resulting in the inability to transport acylated very long chain fatty acids (VLCFAs) into the peroxisome for degradation. VLCFAs subsequently accumulate in tissues, including the central nervous system. Up to 40% of b...
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| Publicado no: | Blood |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6440119/ https://ncbi.nlm.nih.gov/pubmed/30635285 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2018-11-887240 |
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