Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …

Adrenoleukodystrophy (ALD) is a rare X‐linked disease caused by a mutation of the
peroxisomal ABCD1 gene. This review summarizes our current understanding of the …

Pathogenic variants in the ABCD1 gene cause adrenoleukodystrophy (ALD), a progressive
metabolic disorder characterized by 3 core clinical syndromes: a slowly progressive …

Gene transfer into autologous hematopoietic stem progenitor cells (HSPCs) has the
potential to cure monogenic inherited disorders caused by an altered development and/or …

Background Among boys with X‐Linked adrenoleukodystrophy, a subset will develop
childhood cerebral adrenoleukodystrophy (CCALD). CCALD is typically lethal without …

Background X-linked adrenoleukodystrophy (X-ALD) is highly variable, ranging from slowly
progressive adrenomyeloneuropathy to severe brain demyelination and inflammation …

Perinatal white matter injury (WMI) is the most common brain injury in premature infants and
can lead to life-long neurological deficits such as cerebral palsy. Preterm birth is typically …

Adrenoleukodystrophy (ALD) is a rare X-linked disorder of peroxisomal oxidation due to
mutations in ABCD1. It is a progressive condition with a variable clinical spectrum that …

Abstract X-linked adrenoleukodystrophy (X-ALD), the most frequent monogenetic disorder of
brain white matter, is highly variable, ranging from slowly progressive …

Background Adult patients with adrenoleukodystrophy have a poor prognosis owing to
development of adrenomyeloneuropathy. Additionally, a large proportion of patients with …