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filingDate 2019-09-12-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_8ba53c041e0cee2d6c7733b4861f5e23
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publicationDate 2020-04-16-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2020056155-A3
titleOfInvention Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
abstract This disclosure relates to mRNA therapy for the treatment of maple syrup urine disease (MSUD). mRNAs for use in the invention, when administered in vivo, encode branched chain α-ketoacid dehydrogenase complex (BCKDC) E1α, E1β, or E2mRNA therapies of the disclosure increase and/or restore deficient levels of E1α, E1β, or E2 expression and/or BCKDC activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of branched chain amino acids associated with deficient BCKDC activity in subjects.
priorityDate 2018-09-13-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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