Platenburg, Gerard Johannes,Wansink, Derick Gert,De Kimpe, Josephus Johannes
申请号：
AU2007282224
公开号：
AU2007282224B2
申请日:
2007.08.10
申请国别(地区):
AU
年份:
2013
代理人:
摘要：
The current invention provides for methods and medicaments that apply oligonucleotidemolecules complementary onl y to a repetitive sequence in a human gene transcript,for the manufacture of a medicament for the diagnosis, treatment or preventionof a cis-element repeat instability associated genetic disorders in humans.The invention hence provides a method of treatment for cis-element repeat instabilityassociated genetic disorders. The invention also pertains to modified oligonucleotideswhich can be applied in method of the invention to prevent the accumulation and/ortranslation of repeat expanded transcripts in cells.
