PEPTIDE CONJUGATES OF SPLICE-CORRECTING OLIGONUCLEOTIDES FOR ENHANCED CELL AND IN VIVO DELIVERY FOR THERAPY OF NEUROMUSCULAR DISEASES

Grant number: 1034300 | Funding period: 2012 - 2016

Completed

Abstract

Currently, there is no known cure for certain neuromuscular genetic disorders. However, recently identified synthetic DNA-type biomolecules have shown promising results in reversing such diseases in mice. These biomolecules cannot easily enter the cells in high enough quantity to elicit their beneficial effects. Therefore, this project will aim at identifying novel vecotrs that, when coupled to these biomolecules, are capable of delivering them into specific cell types as well as into the brain.