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filingDate 2020-07-17-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_fe167e75fe70e325b551bdfcdffae266
publicationDate 2021-11-18-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber AU-2020316339-A1
titleOfInvention AAV cardiac gene therapy for cardiomyopathy in humans
abstract The present disclosure is related to compositions and methods useful in treating heart conditions. The disclosed compositions and methods are based on gene therapies comprising a recombinant AAV vector for delivering two or more transgenes into the heart of a human subject, wherein the transgenes comprise an S100A1 protein and a cardiac Apoptosis Repressor with caspase recruitment Domain (cARC) apoptotic inhibitor. In various embodiments, the compositions and methods disclosed herein comprise vectors comprising S100A1 and/or cARC cDNA sequences that are codon-optimized for expression in humans. In some aspects, targeting multiple sources of one or more heart conditions can provide synergistic benefits during treatment.
priorityDate 2019-07-19-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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