AAV-mediated gene therapy for metabolic diseases: dosage and reapplication studies in the molybdenum cofactor deficiency model
Author:
Publisher
Springer Science and Business Media LLC
Subject
Molecular Medicine,Immunology,Immunology and Allergy,Biotechnology
Reference18 articles.
1. MOLYBDENUM COFACTOR BIOSYNTHESIS AND MOLYBDENUM ENZYMES
2. Genetics of molybdenum cofactor deficiency
3. Mutations in the molybdenum cofactor biosynthetic genesMOCS1, MOCS2, andGEPH
4. Molybdenum cofactor-deficient mice resemble the phenotype of human patients
5. Long-Term Rescue of a Lethal Inherited Disease by Adeno-Associated Virus–Mediated Gene Transfer in a Mouse Model of Molybdenum-Cofactor Deficiency
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1. Molybdenum cofactor biology, evolution and deficiency;Biochimica et Biophysica Acta (BBA) - Molecular Cell Research;2021-01
2. Delineating the phenotypic spectrum of sulfite oxidase and molybdenum cofactor deficiency;Neurology Genetics;2020-07-14
3. Molybdenum cofactor deficiency type B knock-in mouse models carrying patient-identical mutations and their rescue by singular AAV injections;Human Genetics;2019-02-27
4. Metabolic etiologies in West syndrome;Epilepsia Open;2018-03-14
5. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems;Gene Therapy;2012-01-26
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