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Low donor chimerism may be sufficient to prevent demyelination in adrenoleukodystrophy

Adrenoleukodystrophy (ALD) is a peroxisomal disorder characterized by white matter degeneration caused by adenosine triphosphate‐binding cassette subfamily D member 1 (ABCD1) gene mutations, which lead to an accumulation of very‐long‐chain fatty acids (VLCFA). Hematopoietic stem cell transplantation...

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Detalles Bibliográficos
Autores principales: Ikeda, Takahiro, Kawahara, Yuta, Miyauchi, Akihiko, Niijima, Hitomi, Furukawa, Rieko, Shimozawa, Nobuyuki, Morimoto, Akira, Osaka, Hitoshi, Yamagata, Takanori
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8743339/
https://www.ncbi.nlm.nih.gov/pubmed/35028267
http://dx.doi.org/10.1002/jmd2.12259