CPC A61K 48/005 (2013.01) [A61K 35/761 (2013.01); C12N 9/14 (2013.01); C12N 15/86 (2013.01); C07H 21/04 (2013.01); C12N 15/8645 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01)] | 21 Claims |
1. A method of treating a deficiency in peroxisome biogenesis factor 1 (PEX1) in a subject in need thereof, comprising directly administering to an eye of the subject a recombinant adeno-associated virus (rAAV) comprising an AAV capsid having packaged therein a vector genome comprising a nucleic acid sequence that encodes human PEX1, thereby improving visual function in the subject.
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