ArticleProceedings of the National Academy of Sciences of the United States of America1995
Retroviral-mediated gene transfer corrects very-long-chain fatty acid metabolism in adrenoleukodystrophy fibroblasts.
Article in Proceedings of the National Academy of Sciences of the United States of America, 1995. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed, 104 citations in OpenAlex.
- An AAV-Based Therapy Approach for Neurological Phenotypes of X-Linked Adrenoleukodystrophy.International journal of molecular sciences · 2025Review
- Review
- Peroxisomal ABC Transporters: An Update.International journal of molecular sciences · 2021Review
- Evolution of adrenoleukodystrophy model systems.Journal of inherited metabolic disease · 2021Review
- Acyl-CoA thioesterase activity of peroxisomal ABC protein ABCD1 is required for the transport of very long-chain acyl-CoA into peroxisomes.Scientific reports · 2021Article
- Endocrine dysfunction in adrenoleukodystrophy.Handbook of clinical neurology · 2021Review
- The Landscape of Hematopoietic Stem Cell Transplant and Gene Therapy for X-Linked Adrenoleukodystrophy.Current treatment options in neurology · 2019Review
- ABC Transporter Subfamily D: Distinct Differences in Behavior between ABCD1-3 and ABCD4 in Subcellular Localization, Function, and Human Disease.BioMed research international · 2016Review
- ABCD1 deletion-induced mitochondrial dysfunction is corrected by SAHA: implication for adrenoleukodystrophy.Journal of neurochemistry · 2015Article
- Mammalian peroxisomal ABC transporters: from endogenous substrates to pathology and clinical significance.British journal of pharmacology · 2011Review
- Biochemical aspects of X-linked adrenoleukodystrophy.Brain pathology (Zurich, Switzerland) · 2010Review
- General aspects and neuropathology of X-linked adrenoleukodystrophy.Brain pathology (Zurich, Switzerland) · 2010Review
- Fast diffusion of very long chain saturated fatty acids across a bilayer membrane and their rapid extraction by cyclodextrins: implications for adrenoleukodystrophy.The Journal of biological chemistry · 2009Article
- Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein.Proceedings of the National Academy of Sciences of the United States of America · 2004Article
- Peroxisomal Acyl-CoA synthetase activity is essential for seedling development in Arabidopsis thaliana.The Plant cell · 2004Article
- Peroxisomal disorders: genotype, phenotype, major neuropathologic lesions, and pathogenesis.Brain pathology (Zurich, Switzerland) · 1998Review
- A mouse model for X-linked adrenoleukodystrophy.Proceedings of the National Academy of Sciences of the United States of America · 1997Article
- X linked adrenoleukodystrophy: clinical presentation, diagnosis, and therapy.Journal of neurology, neurosurgery, and psychiatry · 1997Review
- Mutational and protein analysis of patients and heterozygous women with X-linked adrenoleukodystrophy.American journal of human genetics · 1996Article
- A close relative of the adrenoleukodystrophy (ALD) gene codes for a peroxisomal protein with a specific expression pattern.Proceedings of the National Academy of Sciences of the United States of America · 1996Article
Corrections and comments
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Authors and funding
10 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Adrenoleukodystrophy (ALD), a lethal demyelinating disease of the brain, is caused by mutations of a gene encoding an ATP-binding transporter, called ALDP, localized in the peroxisomal membrane. It is associated with a defective oxidation of very-long-chain fatty acids, leading to their accumulation in many tissues. This study reports that the retroviral-mediated transfer of the ALD cDNA restored very-long-chain fatty acid oxidation in ALD fibroblasts in vitro following abundant expression and appropriate targeting of the vector-encoded ALDP in peroxisomes. The same method may be used in hematopoietic cells as a further step of a gene therapy approach of ALD.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.