Methods of treating hearing loss using a secreted target protein
AssigneeAkouos, Inc.
InventorsEmmanuel J. Simons, Robert Ng, Danielle R. Lenz, Hao Chiang
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Abstract
Provided herein are compositions that include a single nucleic acid vector or two different nucleic acid vectors, and the use of these compositions to treat hearing loss in a subject.
Claims33
A construct comprising:
- (i) a cytomegalovirus (CMV) enhancer;
- (ii) a chicken β-actin (CBA) promoter;
- (iii) a chimeric intron comprising the nucleic acid sequence of SEQ ID NO:
- 19;
- (iv) a coding sequence comprising a first nucleic acid sequence encoding a Norrin Cystine Knot Growth Factor (NDP) protein;
- and
- (v) a polyadenylation sequence.
An AAV particle comprising a construct, wherein the construct comprises:
- (i) a cytomegalovirus (CMV) enhancer;
- (ii) a chicken β-actin (CBA) promoter;
- (iii) a chimeric intron comprising the nucleic acid sequence of SEQ ID NO 19;
- (iv) a coding sequence comprising a first nucleic acid sequence encoding a Norrin Cystine Knot Growth Factor (NDP) protein;
- and
- (v) a polyadenylation sequence.
A composition comprising a construct and a pharmaceutically acceptable carrier, diluent or excipient, wherein the construct comprises:
- (i) a cytomegalovirus (CMV) enhancer;
- (ii) a chicken β-actin (CBA) promoter;
- (iii) a chimeric intron comprising the nucleic acid sequence of SEQ ID NO 19;
- (iv) a coding sequence comprising a first nucleic acid sequence encoding a Norrin Cystine Knot Growth Factor (NDP) protein;
- and
- (v) a polyadenylation sequence.
A composition comprising an AAV particle and a pharmaceutically acceptable carrier, diluent or excipient, wherein the AAV particle comprises a construct, wherein the construct comprises:
- (i) a cytomegalovirus (CMV) enhancer;
- (ii) a chicken β-actin (CBA) promoter;
- (iii) a chimeric intron comprising the nucleic acid sequence of SEQ ID NO:
- 19;
- (iv) a coding sequence comprising a first nucleic acid sequence encoding a Norrin Cystine Knot Growth Factor (NDP) protein;
- and
- (v) a polyadenylation sequence.
A kit comprising a composition, wherein the composition comprises (a) a construct or (b) an AAV particle comprising the construct, wherein the construct comprises:
- (i) a cytomegalovirus (CMV) enhancer;
- (ii) a chicken β-actin (CBA) promoter;
- (iii) a chimeric intron comprising the nucleic acid sequence of SEQ ID NO:
- 19;
- (iv) a coding sequence comprising a first nucleic acid sequence encoding a Norrin Cystine Knot Growth Factor (NDP) protein;
- and
- (v) a polyadenylation sequence.
Dependencies are inferred from the claim text.