Oligonucleotide composition; methods for altering the splicing of a transcribed White; and use of the composition for treating Duchenne Muscular Dystrophy.

摘要

Among other things, this Disclosure relates to methods and compositions designed oligonucleotides,.In some forms of realization, the compositions provide a oligonucleotidicas provided altered splicing of the transcript.In some forms of realization, the compositions oligonucleotídicas provided have Low toxicity.In some forms of realization, the compositions provide best Profiles provided oligonucleotídicas binding proteins.In some forms of realization, the compositions oligonucleotidicas provided have better Supply.In some forms of realization, the compositions oligonucleotídicas provided have better reception.In some forms of realization, this Revelation properciona methods for Treatment of diseases using compositions provided oligonucleotídicas.