Aguilera Diez Maria Begoña,De Visser Peter Christian,Mulders Susan Allegonda Maria
申请号：
NZ71339012
公开号：
NZ713390A
申请日:
2012.04.23
申请国别(地区):
NZ
年份:
2017
代理人:
摘要：
Discloses compounds comprising or consisting of the oligonucleotide sequence (NAG)m, wherein N is C or 5-methylcytosine and at least one occurrence of N is 5- methylcytosine and wherein m is 7, 8, 9, 10, 11, 12, 13, 14, or 15 and wherein no inosine nucleotide is present. Also discloses use of compound to treat dystrophy type 1, spino-cerebellar ataxia 8 and/or Huntington’s disease-like 2 caused by expansion of CUG repeats.
