Lutz, Gordon J.,Tallent, Melanie K.,Lykens, Nicole Michele
申请号：
AU2010204639
公开号：
AU2010204639B2
申请日:
2010.01.14
申请国别(地区):
AU
年份:
2014
代理人:
摘要：
The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.
