Catherine Matthys;Michael M. Haley;John Andrew Berglund;Cameron L. Hilton;M. Bryan Warf
发明人:
John Andrew Berglund,M. Bryan Warf,Catherine Matthys,Michael M. Haley,Cameron L. Hilton
申请号：
US12918696
公开号：
US08436049B2
申请日:
2009.02.20
申请国别(地区):
US
年份:
2013
代理人:
摘要：
Methods are provided herein for treatment of myotonic dystrophy and other toxic RNA diseases in a subject. In some examples, the method comprises administration of a compound that binds a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating the disease. In additional examples, the method comprises administration of a compound that disrupts binding of muscleblind-like proteins to an RNA nucleotide repeat expansion. Compounds for use in the disclosed method include pentamidine or heptamidine or derivatives thereof. Representative compounds are described herein.
