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4. Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model. Greig JA; Jennis M; Dandekar A; Chorazeczewski JK; Smith MK; Ashley SN; Yan H; Wilson JM Mol Genet Metab; 2021; 134(1-2):139-146. PubMed ID: 34454844 [TBL] [Abstract][Full Text] [Related]
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