Compositions and methods for treating non-age-associated hearing impairment in a human subject
AssigneeAkouos, Inc.
InventorsEmmanuel John Simons, Robert Ng
Life of this patent
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Abstract
Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject.
Claims28
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.
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.
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.
Dependencies are inferred from the claim text.