COMPOSITIONS AND METHODS FOR DELIVERING THERAPEUTIC POLYNUCLEOTIDES FOR EXON SKIPPING

Abstract: compositions and methods are provided for delivering an antisense oligonucleotide amendable to exon skipping by administering a complex formed between an antisense oligonucleotide and a 3e10 antibody or antigen-binding fragment thereof. in some instances, the complexes are stabilized and demonstrate cellular localization through a molar ratio of 3e10 antibody or antigen-binding fragment thereof to antisense oligonucleotide of at least about 2:1.

Inventor(s): Elias Quijano, Peter Glazer, Stephen Squinto, Dale Ludwig, Bruce Turner, Josage Dinithi Perera

CPC Classification: A61K47/6807 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

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