Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy

Abstract excerpt

Adrenoleukodystrophy (ALD) is a rare neurometabolic disease caused by mutations in the ABCD1 gene, which encodes for the peroxisomal very long chain fatty acid (VLCFAs) transporter. It is a debilitating disorder, which has a spectrum of clinical presentations. The most severe form is a rapidly progressing demyelinating disease called cerebral ALD or CALD. Patients with cALD have a life expectancy of 2-4 years aft...

Topics

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• Adrenoleukodystrophy
• ATP Binding Cassette Transporter, Subfamily D, Member 1
• Disease Models, Animal
• Genetic Therapy
• Hematopoietic Stem Cell Transplantation
• Mice
• Mutation
• Peroxisome Proliferator-Activated Receptors

Identifiers and source

Literature Corpus work

9600ebbe-5e86-5e1c-b93b-9f3466ba5c5a

DOI

10.1101/2024.11.11.622837