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US 12,264,317 B2Granted
Compositions and methods for treating non-age-associated hearing impairment in a human subject
AssigneeAkouos, Inc.
InventorsEmmanuel John Simons, Ellen Reisinger, Sebastian Kügler, Hanan Al-Moyed
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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.
Claims30
A method of treating otoferlin related hearing loss in a subject, comprising administering to the subject a plurality of AAV vectors comprising:
- a) a first AAV vector comprising a 5′ inverted terminal repeat (ITR), a 3′ ITR, a F1 phage recombinogenic region, and a promoter operably linked to a portion of a gene encoding an otoferlin polypeptide;
- and
- b) a second AAV vector comprising a 5′ ITR, a 3′ ITR, a F1 phage recombinogenic region, and a portion of the gene encoding an otoferlin polypeptide;
- wherein the first AAV vector and the second AAV vector are capable of constituting a full-length otoferlin messenger RNA in the subject;
- wherein the first and second AAV vectors each include a total number of nucleotides of up to about 5kb;
- and
- wherein the otoferlin related hearing loss is associated with reduced expression, lack of expression, or dysfunction of otoferlin.
4 more elements
A method of treating otoferlin related hearing loss in a subject, comprising administering to the subject a plurality of AAV vectors comprising:
- a) a first AAV vector comprising (i) a promoter, (ii) a portion of a gene encoding an otoferlin polypeptide, (iii) a splicing donor signal sequence, (iv) a 77 base pair F1 phage recombinogenic region, and (v) 5′ and 3′ ITRs;
- and
- b) a second AAV vector comprising (i) a 77 base pair F1 phage recombinogenic region, (ii) a splicing acceptor signal sequence, (iii) a portion of the gene encoding an otoferlin polypeptide, (iv) a polyadenylation signal, and (v) 5′ and 3′ ITRs;
- wherein the first AAV vector and the second AAV vector are capable of constituting a full-length otoferlin messenger RNA in the subject;
- wherein the first and second AAV vectors each include a total number of nucleotides of up to about 5 kb;
- and
- wherein the otoferlin related hearing loss is associated with reduced expression, lack of expression, or dysfunction of otoferlin.
4 more elements
Dependencies are inferred from the claim text.