ArticleMolecular genetics and metabolism2015
Correction of a genetic deficiency in pantothenate kinase 1 using phosphopantothenate replacement therapy.
Article in Molecular genetics and metabolism, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 36 citations in OpenAlex.
- A liquid chromatography-mass spectrometry method to quantify total Coenzyme A concentration and isotopic labeling.bioRxiv : the preprint server for biology · 2026Article
- A therapeutic approach to pantothenate kinase associated neurodegeneration: a pilot study.Orphanet journal of rare diseases · 2024Article
- Patient-Derived Cellular Models for Polytarget Precision Medicine in Pantothenate Kinase-Associated Neurodegeneration.Pharmaceuticals (Basel, Switzerland) · 2023Review
- Inherited Disorders of Coenzyme A Biosynthesis: Models, Mechanisms, and Treatments.International journal of molecular sciences · 2023Review
- Generation and Validation of an Anti-Human PANK3 Mouse Monoclonal Antibody.Biomolecules · 2022Article
- Therapeutic approach with commercial supplements for pantothenate kinase-associated neurodegeneration with residual PANK2 expression levels.Orphanet journal of rare diseases · 2022Article
- Malonyl-acyl carrier protein decarboxylase activity promotes fatty acid and cell envelope biosynthesis in Proteobacteria.The Journal of biological chemistry · 2021Article
- Pantothenate kinase activation relieves coenzyme A sequestration and improves mitochondrial function in mice with propionic acidemia.Science translational medicine · 2021Article
- Turnover rate of coenzyme A in mouse brain and liver.PloS one · 2021Article
- A long hypoxia-inducible factor 3 isoform 2 is a transcription activator that regulates erythropoietin.Cellular and molecular life sciences : CMLS · 2020Article
- Metabolomic analysis of primary human skeletal muscle cells during myogenic progression.Scientific reports · 2020Article
- A pantothenate kinase-deficient mouse model reveals a gene expression program associated with brain coenzyme a reduction.Biochimica et biophysica acta. Molecular basis of disease · 2020Article
- Phosphonate prodrugs: an overview and recent advances.Future medicinal chemistry · 2019Review
- Human pantothenate kinase 4 is a pseudo-pantothenate kinase.Protein science : a publication of the Protein Society · 2019Article
- Proposed Therapies for Pantothenate-Kinase-Associated Neurodegeneration.Journal of experimental neuroscience · 2019Article
- A therapeutic approach to pantothenate kinase associated neurodegeneration.Nature communications · 2018Article
- Article
- Acetyl-4'-phosphopantetheine is stable in serum and prevents phenotypes induced by pantothenate kinase deficiency.Scientific reports · 2017Article
- Excess coenzyme A reduces skeletal muscle performance and strength in mice overexpressing human PANK2.Molecular genetics and metabolism · 2017Article
- Open-Label Fosmetpantotenate, a Phosphopantothenate Replacement Therapy in a Single Patient with Atypical PKAN.Case reports in neurological medicine · 2017Article
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Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
Coenzyme A (CoA) is a ubiquitous cofactor involved in numerous essential biochemical transformations, and along with its thioesters is a key regulator of intermediary metabolism. Pantothenate (vitamin B5) phosphorylation by pantothenate kinase (PanK) is thought to control the rate of CoA production. Pantothenate kinase associated neurodegeneration is a hereditary disease that arises from mutations that inactivate the human PANK2 gene. Aryl phosphoramidate phosphopantothenate derivatives were prepared to test the feasibility of using phosphopantothenate replacement therapy to bypass the genetic deficiency in the Pank1(-/-) mouse model. The efficacies of candidate compounds were first compared by measuring the ability to increase CoA levels in Pank1(-/-) mouse embryo fibroblasts. Administration of selected candidate compounds to Pank1(-/-) mice corrected their deficiency in hepatic CoA. The PanK bypass was confirmed by the incorporation of intact phosphopantothenate into CoA using triple-isotopically labeled compound. These results provide strong support for PanK as a master regulator of intracellular CoA and illustrate the feasibility of employing PanK bypass therapy to restore CoA levels in genetically deficient mice.
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Registered trials
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.