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Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets

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    In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

    In utero base editing has the potential to correct disease-causing mutations before the onset of pathology. Mucopolysaccharidosis type I (MPS-IH, Hurler syndrome) is a lysosomal storage disease (LSD) affecting...

    Sourav K. Bose, Brandon M. White, Meghana V. Kashyap, Apeksha Dave… in Nature Communications (2021)

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