Publication | Open Access

Genetic Basis for Correction of Very-Long-Chain Acyl–Coenzyme A Dehydrogenase Deficiency by Bezafibrate in Patient Fibroblasts: Toward a Genotype-Based Therapy

Genetic DiseasesGene TherapiesGenotype-based TherapyMendelian DisorderGenetic DisorderGeneticsInherited Metabolic DiseasePathologyMolecular GeneticsDisease Gene IdentificationPatient FibroblastsMedicineGenomic MedicineGenetic BasisMonogenic Disorders