X-linked adrenoleukodystrophy is a severe progressive neurological disease that is predominantly found in male patients and caused by mutations in the X-linked ABCD1 gene encoding peroxisome transport protein. The disease is clinically characterized by two main phenotypes: the most severe infant cerebral form and adrenomyeloneuropathy. The disease is treated by allogeneic transplantation of hematopoietic cells from a healthy donor to stop progression, and gene therapy with a self-activating lentiviral vector, the carrier of the functional gene ABCD1. Each method has its own limitations. The authors present and theoretically substantiate an alternative approach to the treatment of adrenoleukodystrophy; they propose to modify the autologous C...
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and adrenal cortex sec...
WOS: 000340492600028PubMed ID: 24788897X linked adrenoleukodystrophy (X-ALD) is a neurodegenerative ...
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and adrenal cortex sec...
X-linked adrenoleukodystrophy (X-ALD) is caused by mutations in the ATP binding cassette subfamily D...
International audienceX-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in...
Adrenoleukodystrophy (ALD) is caused by various pathogenic mutations in the X-linked ABCD1 gene, whi...
International audienceX-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in...
X-linked adrenoleukodystrophy (X-ALD) is an inherited neurodegenerative disorder associated with mut...
BackgroundIn X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD p...
X-linked adrenoleukodystrophy (ALD) caused by the ABCD1 mutation, is the most common inherited perox...
Mutations in the ABCD1 gene cause the clinical spectrum of the neurometabolic disorder X-linked adre...
Adrenoleukodystrophy (ALD) is a rare X-linked disorder of peroxisomal oxidation due to mutations in ...
Adrenoleukodystrophy (ALD) is an X-linked genetic disorder, characterized by demyelination in the ce...
Leukodystrophies (LDs) refer to a group on inherited diseases in which molecular abnormalities of gl...
Abstract Objectives X-linked adrenoleukodystrophy is a neurodegenerative disorder caused by mutation...
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and adrenal cortex sec...
WOS: 000340492600028PubMed ID: 24788897X linked adrenoleukodystrophy (X-ALD) is a neurodegenerative ...
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and adrenal cortex sec...
X-linked adrenoleukodystrophy (X-ALD) is caused by mutations in the ATP binding cassette subfamily D...
International audienceX-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in...
Adrenoleukodystrophy (ALD) is caused by various pathogenic mutations in the X-linked ABCD1 gene, whi...
International audienceX-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in...
X-linked adrenoleukodystrophy (X-ALD) is an inherited neurodegenerative disorder associated with mut...
BackgroundIn X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD p...
X-linked adrenoleukodystrophy (ALD) caused by the ABCD1 mutation, is the most common inherited perox...
Mutations in the ABCD1 gene cause the clinical spectrum of the neurometabolic disorder X-linked adre...
Adrenoleukodystrophy (ALD) is a rare X-linked disorder of peroxisomal oxidation due to mutations in ...
Adrenoleukodystrophy (ALD) is an X-linked genetic disorder, characterized by demyelination in the ce...
Leukodystrophies (LDs) refer to a group on inherited diseases in which molecular abnormalities of gl...
Abstract Objectives X-linked adrenoleukodystrophy is a neurodegenerative disorder caused by mutation...
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and adrenal cortex sec...
WOS: 000340492600028PubMed ID: 24788897X linked adrenoleukodystrophy (X-ALD) is a neurodegenerative ...
X-linked adrenoleukodystrophy (X-ALD) affects the nervous system white matter and adrenal cortex sec...