CPC C12N 15/86 (2013.01) [A61P 27/16 (2018.01); C07K 14/47 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14144 (2013.01)] | 28 Claims |
1. A plurality of recombinant adeno-associated viral (rAAV) vectors comprising:
a) a first rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 96; and
b) a second rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 105.
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10. A method of expressing a recombinant full-length otoferlin protein in a mammalian cell, the method comprising administering a plurality of recombinant adeno-associated viral (rAAV) vectors comprising:
a) a first rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 96; and
b) a second rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 105;
to the mammalian cell, wherein the mammalian cell has reduced expression, lack of expression or dysfunction of otoferlin.
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19. A method of treating hearing loss in a subject having otoferlin related hearing loss, the method comprising administering a plurality of recombinant adeno-associated viral (rAAV) vectors comprising:
a) a first rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 96; and
b) a second rAAV vector comprising a nucleic acid sequence of SEQ ID NO: 105;
into the cochlea of the subject, wherein the subject has reduced expression, lack of expression, or dysfunction of otoferlin.
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