公开(公告)号：US20150275211A1

公开(公告)日：2015-10-01

申请号：US14440018

申请日：2013-10-31

Applicant: ISIS PHARMACEUTICALS, INC. ,  COLD SPRING HARBOR LABORATORY

Inventor： Frank Rigo ,  C. Bennett ,  Adrian R. Krainer ,  Zhenxun Wang

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/14 ,  C12Y207/0104

Abstract: The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript. Certain such compounds are useful for hybridizing to a pyruvate kinase M transcript, including but not limited to a pyruvate kinase M transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the pyruvate kinase M transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with cancer.

Abstract translation: 本发明提供了包含与丙酮酸激酶M转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与丙酮酸激酶M转录物杂交，包括但不限于细胞中的丙酮酸激酶M转录物。 在某些实施方案中，这种杂交导致丙酮酸激酶M转录物剪接的调节。 在某些实施方案中，这些化合物用于治疗与癌症相关的一种或多种症状。