Title:
USE OF ADENO-ASSOCIATED VIRAL VECTORS TO CORRECT GENE DEFECTS/ EXPRESS PROTEINS IN HAIR CELLS AND SUPPORTING CELLS IN THE INNER EAR
Document Type and Number:
WIPO Patent Application WO/2020/097372
Kind Code:
A8
Abstract:
Provided herein are compositions that include at least two different nucleic acid vectors that, when introduced into a primate cell, the at least two different vectors undergo concatamerization or homologous recombination with each other, thereby forming a recombined nucleic acid that encodes a full-length target protein (e.g., a supporting cell target protein or a hair cell target protein). Also provided are compositions that include a single AAV vector that, when introduced into a primate cell, a nucleic acid encoding a full-length target protein (e.g., a supporting cell target protein or a hair cell target protein) is generated at the locus of the supporting cell target gene, and the primate expresses the target protein (e.g., a supporting cell target protein or a hair cell target protein).
Inventors:
SIMONS EMMANUEL J (US)
NG ROBERT (US)
LENZ DANIELLE (US)
VALERO MICHELLE (US)
NG ROBERT (US)
LENZ DANIELLE (US)
VALERO MICHELLE (US)
Application Number:
PCT/US2019/060328
Publication Date:
April 29, 2021
Filing Date:
November 07, 2019
Export Citation:
Assignee:
AKOUOS INC (US)
International Classes:
C12N15/86; A61K48/00; A61P27/16; C07K14/47; C07K14/705; C12N15/113; C12N15/65
Attorney, Agent or Firm:
LYON, Charles E. et al. (US)
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