Josephus Johannes De Kimpe,Gerard Johannes Platenburg,Derick Gert Wansink
申请号：
US15855848
公开号：
US10689646B2
申请日:
2017.12.27
申请国别(地区):
US
年份:
2020
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.
