AGUILERA DIEZ MARIA BEGOÑA,DE VISSER PETER CHRISTIAN,MULDERS SUSAN ALLEGONDA MARIA
申请号：
NZ61676212
公开号：
NZ616762A
申请日:
2012.04.23
申请国别(地区):
NZ
年份:
2015
代理人:
摘要：
Disclosed is a compound comprising a peptide part comprising LGAQSNF linked to an oligonucleotide part comprising (NAG)m in which N is C or 5-methylcytosine, and wherein m is 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, or 15. Also disclosed is the use of such a compound for the manufacture of a medicament for treating, preventing and/or delaying dystrophy type 1 (DM1), spino-cerebellar ataxia 8 and/or Huntington&rsquos disease-like 2 caused by expansion of CUG repeats in transcripts of the DM1/DMPK, SCA8 or JPH3 genes.
