Exploratory study of autophagy inducer sirolimus for childhood cerebral adrenoleukodystrophy

• Main Authors: Xiao-Mei Luo (Author), Li-Ying Liu (Author), Qiu-Hong Wang (Author), Yang-Yang Wang (Author), Jing Wang (Author), Xiao-Yan Yang (Author), Shi-Jun Li (Author), Li-Ping Zou (Author)
• Format: Book
• Published: Frontiers Media S.A., 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

ObjectivesX-linked adrenoleukodystrophy (ALD) is a peroxisomal disease caused by mutations in the ABCD1 gene. Childhood cerebral ALD (CCALD) is characterized by inflammatory demyelination, rapidly progressing, often fatal. Hematopoietic stem cell transplant only delays disease progression in patient...