AAV vectors encoding mini-PCDH15 and uses thereof
AssigneesPresident and Fellows of Harvard College, Ohio State Innovation Foundation, Massachusetts Eye and Ear Infirmary
InventorsDavid P. Corey, Artur Indzhykulian, Marcos Sotomayor, Maryna V. Ivanchenko, Cole W. D. Peters
Life of this patent
Estimating the term…
Abstract
Aspects of the disclosure relate to compositions, nucleic acids, vectors, viruses, and methods useful for treating hearing loss and/or blindness, for example, Usher Syndrome type IF. The present disclosure provides isolated nucleic acids, vectors, and rAAV.9.PHP.B comprising a transgene encoding a mini-PCDH15, and methods of treating hearing loss using the same. The present disclosure also provides a gRNA associated with a base editor to correct one or more mutations in PCDH15 for treating hearing loss and/or vision loss.
Claims15
An isolated nucleic acid comprising a transgene flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs), wherein the transgene encodes a mini-Protocadherin related 15 (mini-PCDH15) protein comprising an amino acid sequence at least 95% identical to SEQ ID NO:
- 95 or 99.
Dependencies are inferred from the claim text.