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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| Auteurs principaux: | , , , , , , , |
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| Format: | article |
| Langue: | EN |
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Public Library of Science (PLoS)
2014
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| Sujets: | |
| Accès en ligne: | https://doaj.org/article/3cd89c0996e947dbad4aacf79f64a7c4 |
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