Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing
Leah E. Venturoni, Randy J. Chandler, Jing Liao, Victoria Hoffmann, Nikhil Ramesh, Susana Gordo, Nelson Chau, Charles P. Venditti
Topics & Concepts
Methylmalonic acidemiaGenome editingExonGenetic enhancementBiologyNucleaseGeneTranscription activator-like effector nucleaseAdeno-associated virusTransgeneGeneticsMolecular biologyGenomeEndocrinologyVector (molecular biology)Recombinant DNAMetabolism and Genetic DisordersVirus-based gene therapy researchDiabetes and associated disorders