Methods 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
Life of this patent
Estimating the term…
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.
Claims23
A guide RNA (gRNA) comprising a nucleic acid sequence having at least 75% identity to any one of SEQ ID NOs:
- 21-33 and 90-95.
A chimeric molecule comprising a supercharged protein domain, a gene editing domain, and a membrane destabilizing protein domain.
Dependencies are inferred from the claim text.