专利检索 ap:("The Regents of the University of Colorado, a body corporate") AND inv:"John Rinn" 第 1 页

1.

发明公开
Method for Retaining Splicing RNAs in the Nucleus Based on Chemically Modified Antisense Oligonucleotides 审中-公开

公开(公告)号：US20230257741A1

公开(公告)日：2023-08-17

申请号：US17759402

申请日：2021-01-29

申请人： The Regents of the University of Colorado, a body corporate

发明人： Gabrijela Dumbovic , John Rinn , Marvin Caruthers , Katarzyna Jastrzebska , Heera K. Langner

IPC分类号： C12N15/113 , A61K31/7115 , A61P35/00

CPC分类号： C12N15/113 , A61K31/7115 , A61P35/00

摘要： The inventive technology relates to systems and methods for the use of chemically modified antisense oligonucleotides (ASOs) to sterically prevent intron excision thus causing nuclear retention of the RNA and inhibiting nuclear export. In preferred therapeutic embodiments, ASOs may be engineered to quarantine clinically relevant RNAs in the nucleus of the cell.