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Engineering hematopoietic stem cell with ABCD1 to enhance the treatment of X-Linked Adrenoleukodystrophy. Brain MRIs from A) P1 and B) P2 before and after gene therapy. C) Progression of cerebral demyelinating lesions in an untreated 8-year-old ALD patient. Reproduced with permission.[340] Copyright 2009, Science.
