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专利名称：Treatment of genetic disorders associated
with DNA repeat instability
发明人：Josephus Johannes De Kimpe,Gerard
Johannes Platenburg,Derick Gert Wansink
申请号：US14809483申请日：20150727公开号：US090379B2公开日：20180213
摘要：The current invention provides for methods and medicaments that applyoligonucleotide molecules complementary only to a repetitive sequence in a human genetranscript, for the manufacture of a medicament for the diagnosis, treatment or
prevention of a cis-element repeat instability associated genetic disorders in humans. Theinvention 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.
申请人：BioMarin Technologies B.V.
地址：Leiden NL
国籍：NL
代理机构：Jones Day
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