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Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)

Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT),...

Published: 10/28/2024 | Inventor(s): Colin Shea, Ciprian Crainiceanu, Elizabeth Sweeney, Russell Shinohara, Arthur Goldsmith, Daniel Reich

Keywords(s): Automatic, brain, Detection, IMAGING, Incidence, Lesion, Listed LPM Shmilovich as of 4/15/2015, Longitudinal, Magnetic, MULTIMODALITY, Post LPM Assignment Set 20150420, Pre LPM working set 20150418, Resonance, software, SuBLIME:, VPXXXX, WJXXXX, XEXXXX

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