Article

Successful Correction of ALD Patient-derived iPSCs Using CRISPR/Cas9

2020-02-25

Abstract excerpt

X-linked adrenoleukodystrophy (ALD) caused by the ABCD1 mutation, is the most common inherited peroxisomal disease. It is characterized by three phenotypes: inflammatory cerebral demyelination, progressive myelopathy, and adrenal insufficiency, but there is no genotype-phenotype correlation. Hematopoietic stem cell transplantation can only be used in a few patients in the early phase of cerebral inflammation; the...

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Literature Corpus work: 71e1252f-3dea-591b-82a0-a228b14f3e29
DOI: 10.1101/2020.02.23.962118

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