Method for efficient delivery of therapeutic molecules in vitro and in vivo
AssigneeMassachusetts Eye and Ear Infirmary
InventorZheng-Yi Chen
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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.
Claims50
A method of treating deafness or disorders thereof, associated with a genetic mutation in a patient in need thereof, comprising:
- administering to an inner ear of the patient a therapeutically effective amount of a chimeric molecule comprising a membrane destabilizing domain and at least one protein or peptide fused, complexed or linked to one or more one or more oligonucleotides, polynucleotides, gene editing agents, transcriptional modulators, translational modulators, post-translational modulators, and/or modulators that regulate gene expression, protein expression, function, activity or combinations thereof.
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A method of delivering a therapeutic molecule to cells of an inner ear of a patient, comprising:
- administering to the inner ear of a patient a chimeric molecule encapsulated in a cationic lipid formulation, the chimeric molecule comprising at least one protein or peptide fused, complexed or linked to one or more anionic molecules, wherein the protein or peptide comprises an antimicrobial or membrane destabilizing protein or peptides, a supercharged protein or peptide or combinations thereof.
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Dependencies are inferred from the claim text.