| CPC C07K 14/505 (2013.01) [A61K 31/7088 (2013.01); A61K 31/7105 (2013.01); A61K 45/06 (2013.01); A61K 48/005 (2013.01); C12N 15/113 (2013.01); A61K 38/00 (2013.01); A61K 48/00 (2013.01); C12N 2310/531 (2013.01)] | 21 Claims |
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1. A method of treating or preventing propionic acidemia wherein, the method comprises administering to a subject in need thereof an effective amount of
a purified RNA comprising at least one coding sequence encoding a PCCA protein that is at least 95% identical to the amino acid sequence of SEQ ID NO: 8175, wherein the coding sequence comprises the nucleic acid sequence of any one of SEQ ID NOs: 34289, 47346, 60403, 21232 and 73460 or a sequence at least 80% identical to the nucleic acid sequence of any one of SEQ ID NOs: 34289, 47346, 60403, 21232 and 73460.
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