Evaluation of Retinoids for Induction of the Redundant Gene ABCD2 as an Alternative Treatment Option in X-Linked Adrenoleukodystrophy

X-linked adrenoleukodystrophy (X-ALD), the most common peroxisomal disorder, is a clinically heterogeneous disease that can manifest as devastating inflammatory cerebral demyelination (CALD) leading to death of affected males. Currently, the only curative treatment is allogeneic hematopoietic stem c...

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Autori principali: Weber, Franziska D., Weinhofer, Isabelle, Einwich, Angelika, Forss-Petter, Sonja, Muneer, Zahid, Maier, Harald, Weber, Willi H. A., Berger, Johannes
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2014
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4117577/
https://ncbi.nlm.nih.gov/pubmed/25079382
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0103742
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