Adenoassociated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy

X-linked adrenoleukodystrophy (X-ALD) is a devastating neurological disorder caused by mutations in the ABCD1 gene that encodes a peroxisomal ATP-binding cassette transporter (ABCD1) responsible for transport of CoA-activated very long-chain fatty acids (VLCFA) into the peroxisome for degradation. W...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Gong, Yi, Mu, Dakai, Prabhakar, Shilpa, Moser, Ann, Musolino, Patricia, Ren, JiaQian, Breakefield, Xandra O, Maguire, Casey A, Eichler, Florian S
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4427888/
https://ncbi.nlm.nih.gov/pubmed/25592337
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.6
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