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Stem cell-transplantation therapy for adrenoleukodystrophy, reviewed - Cord Blood

Cord Blood

and Stem Cell Banking

Stem cell-transplantation therapy for adrenoleukodystrophy, reviewed

woman holding laboratory appratus

A review has been published about adrenoleukodystrophy (ALD), a rare, X-linked disorder of impaired very long-chain fatty acid metabolism. It focuses on the cerebral variant (cALD), which affects 35% of ALD patients in boyhood and causes rapid neurologic decline. It reviews the current perspectives of stem cell transplantation in cALD, which is the standard of care for early cALD but has less favorable outcomes for advanced disease.

It covers topics such as: the biochemical defect and diagnosis of ALD, the pathophysiology and clinical features of cALD, the history and mechanism of stem cell transplantation in cALD, the outcomes and predictors of stem cell transplantation in cALD, the challenges and future directions of stem cell transplantation in cALD, and the conclusion and recommendations for clinical practice. Keywords include, stem cell transplantation, bone marrow transplantation, umbilical cord-blood transplantation, and hematopoietic cell transplantation.