Methods of treating non-syndromic sensorineural hearing loss
AssigneeAkouos, Inc.
InventorsEmmanuel John Simons, Robert Ng
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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 a stereocilin protein, and the use of these compositions to treat non-syndromic sensorineural hearing loss in a subject.
Claims20
-66. (canceled)
A method of increasing expression of a full-length stereocilin protein in a human cell, the method comprising introducing a composition into the human cell, wherein the composition comprises a first nucleic acid vector and a second nucleic acid vector, wherein:
- the first nucleic acid vector comprises (i) a promoter and (ii) a 5′ coding sequence of a stereocilin protein according to SEQ ID NO:
- 25, and
- the second nucleic acid vector comprises a 3′ coding sequence of a stereocilin protein according to SEQ ID NO:
- 28.
A method of treating non-symptomatic sensorineural hearing loss in a human subject in need thereof, wherein the human subject has been identified as having a defective stereocilin gene, the method comprising:
- administering a therapeutically effective amount of a composition into the cochlea of the human subject, wherein the composition comprises a first nucleic acid vector and a second nucleic acid vector, wherein:
- the first nucleic acid vector comprises (i) a promoter and (ii) a 5′ coding sequence of a stereocilin protein according to SEQ ID NO:
- 25, and
- the second nucleic acid vector comprises a 3′ coding sequence of a stereocilin protein according to SEQ ID NO:
- 28.
A kit comprising a composition that comprises a first nucleic acid vector and a second nucleic acid vector, wherein:
- the first nucleic acid vector comprises (i) a promoter and (ii) a 5′ coding sequence of a stereocilin protein according to SEQ ID NO:
- 25, and
- the second nucleic acid vector comprises a 3′ coding sequence of a stereocilin protein according to SEQ ID NO:
- 28.
Dependencies are inferred from the claim text.