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publicationDate 2018-11-15-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2018144709-A3
titleOfInvention Gene therapy for treating citrullenemia
abstract Compositions and regimens useful in treating type I citrullenemia are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Argininosuccinate Synthase 1 (ASS1).
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