IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA
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United States of America
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Feb 13, 2025
app pub date -
Dec 29, 2022
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Dec 29, 2022
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Abstract
Polynucleotide expression cassettes comprising synthetic polynucleotides encoding human propionyl-CoA carboxylase alpha (synPCCA) are described herein. Related recombinant expression vectors, recombinant adeno-associated viruses (rAAVs), and compositions are also described. Also described are methods of treating a disease or condition mediated by propionyl-CoA carboxylase, comprising administering to a subject in need thereof a therapeutic amount of any of the polynucleotide expression cassettes, recombinant expression vectors, rAAVs, or compositions.
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