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or ald.,

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It is caused by mutations in the ABCD1 gene located on the X-chromosome that encodes the peroxisomal membrane protein ALDP, which is involved in the transmembrane transport of very long-chain fatty acids (VLCFA); therefore, a defect in ALDP results in elevated levels of VLCFA in the plasma and tissues [62].
Professor Aurora Pujol, a research professor for the Catalan Government Research Body ICREA, working as Director of the Neurometabolic Diseases Laboratory at IDIBELL, Barcelona, Spain, investigated the role of mitochondria, the power plant of the cell, in adrenoleukodystrophy, a disease caused by the inactivation of the ABCD1 transporter of fatty acids in peroxisomes.
Adrenolokodistrofi (ALD) X'e bagli otozomal resesif gecis gosteren, peroksizomal membran proteinlerini kodlayan ABCD1 geninin Xq28 kolundaki defektin yol actigi, santral sinir sistemini ve adrenal korteksi etkileyen norodejeneratif bir bozukluktur (1).
ALD is caused by a mutation of the ABCD1 gene and has been mapped to chromosome Xq28.
Lenti-D gene therapy is comprised of a patient's own immature bone marrow cells that are modified to include a functional copy of the ABCD1 gene to express functional ALD protein, which is lacking in patients with CALD.