Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model
Jenny A. Greig, Matthew Jennis, Aditya Dandekar, Joanna K Chorazeczewski, Melanie K. Smith, Scott N. Ashley, Hanying Yan, James M. Wilson
Topics & Concepts
Maple syrup urine diseaseBiologyGenetic enhancementPhenotypeTransplantationEndocrinologyInternal medicineAmino acidBiochemistryGeneMedicineLeucineMetabolism and Genetic DisordersBiochemical and Molecular ResearchMuscle metabolism and nutrition