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Liver-Directed Gene Therapy Shows Durable Effect in MMA Mouse Model
A single systemic dose of an immune-shielded lentiviral vector delivering the MMUT gene produced lasting metabolic correction in a mouse model of methylmalonic acidemia, according to newly published preclinical data.
- Published by
- CGTLive
- Evidence type
- Animal
A single systemic dose of an immune-shielded lentiviral vector delivering the MMUT gene produced lasting metabolic correction in a mouse model of methylmalonic acidemia, according to newly published preclinical data.
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