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Article

Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy

2 Sept 2022

Abstract excerpt

INTRODUCTION: gene. By adulthood, approximately 40% of the patients develop cerebral ALD, a severe, neuroinflammatory condition that is generally progressive and fatal without intervention. AREAS COVERED: Historically, only allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to halt progression of cerebral ALD, with superior outcomes obtained when HSCT is performed early in the disease...

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PMID: 36107226
DOI: 10.1080/14712598.2022.2124857

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