Harvard College

Massachusetts Eye and Ear Infirmary

Margie Li

David R Liu

David B Thompson

John Zuris

Zheng-Yi Chen

David Thompson

David Liu

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.

Methods for efficient delivery of therapeutic molecules in vitro and in vivo

Compositions and provided to induce cells of the inner ear to renter the cell cycle and to proliferate. In particular, hair cells are induced to proliferate by administration of a composition which activates the Myc and Notch. Supporting cells are induced to transdifferentiate to hair cells by inhibition of Myc and Notch activity or the activation of Atoh1. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.

Efficient delivery of therapeutic molecules to cells of the inner ear

Method for efficient delivery of therapeutic molecules in vitro and in vivo

Growing community of inventors

Cambridge, MA, United States of America

Margie Li