GENE THERAPY FOR TREATING PROPIONIC ACIDEMIA
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United States of America Patent
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Issued Date -
Apr 27, 2023
app pub date -
Mar 30, 2021
filing date -
Mar 31, 2020
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Abstract
This present disclosure provides recombinant adeno-associated virus (rAAV) and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of PA caused by a mutation in propionyl-CoA carboxylase α-subunit (PCCA) or a mutation in propionyl-CoA carboxylase β-subunit (PCCB).
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