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Gene Therapy for Cobalamin C Deficiency (cblC) with Viable Mouse Models

Cobalamin C deficiency (cblC) is the most common inborn error of intracellular cobalamin metabolism and is caused by mutations in MMACHC, a gene responsible for processing and trafficking dependent enzymes: intracellular cobalamin, resulting in elevated methylmalonic acid and homocysteine and methionine deficiency. Disease manifestations include growth...

Published: 7/25/2024 | Inventor(s): Jennifer Sloan, Charles Venditti

Keywords(s): C, cblC, Cobalamin, DEFICIENCY, Gene, THERAPY, VDXXXX, VEXXXX, VPXXXX, WIXXXX, WJXXXX, XEXXXX

Category(s): TherapeuticArea > Dental, TherapeuticArea > Endocrinology, Application > Therapeutics, TherapeuticArea > Oncology, TherapeuticArea > Cardiology, TherapeuticArea > Ophthalmology, TherapeuticArea > Neurology, TherapeuticArea > Infectious Disease, Application > Research Materials