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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
BACKGROUND: In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allo...
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| Udgivet i: | N Engl J Med |
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| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2017
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5708849/ https://ncbi.nlm.nih.gov/pubmed/28976817 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1700554 |
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