JOSEPHUS JOHANNES DE KIMPE,GERARDUS JOHANNES PLATENBURG,DERICK GERT WANSINK
申请号：
PT07808532
公开号：
PT2049664E
申请日:
2007.08.10
申请国别(地区):
PT
年份:
2012
代理人:
摘要：
The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
