Method for efficient delivery of therapeutic molecules in vitro and in vivo
AssigneesMassachusetts Eye and Ear Infirmary, President and Fellows of Harvard College
InventorsZheng-Yi Chen, David R. Liu, Margie Li, David B. Thompson, John Zuris
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Abstract
Compositions are described for direct protein delivery into multiple cell types in the mammalian inner ear. The compositions are used to deliver protein(s) (such as gene editing factors) editing of genetic mutations associated with deafness or associated disorders thereof. The delivery of genome editing proteins for gene editing and correction of genetic mutations protect or restore hearing from genetic deafness. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.
Claims11
A method of treating deafness, associated with an autosomal dominant genetic mutation in a TMC1 gene in a patient in need thereof, comprising:
- administering to an inner ear of the patient a therapeutically effective amount of a chimeric molecule consisting of a membrane destabilizing domain, a gene editing agent, wherein the gene editing agent is Cas9, and a gRNA targeting a TMC1 gene, thereby treating deafness.
Dependencies are inferred from the claim text.