公开(公告)号：US11512312B2

公开(公告)日：2022-11-29

申请号：US16492522

申请日：2018-03-09

Applicant: The Board of Regents of the University of Texas System

Inventor： Venkateswara V. Mootha ,  David R. Corey ,  Jiaxin Hu

IPC: C07H21/04 ,  C12N15/113 ,  A61K9/00 ,  A61K31/7105 ,  C12N15/11

Abstract: Described are compounds and methods useful in the treatment of Fuchs' Endothelial Corneal Dystrophy (FECD).

公开(公告)号：US09340785B2

公开(公告)日：2016-05-17

申请号：US14531203

申请日：2014-11-03

Applicant: The Board of Regents of the University of Texas System

Inventor： David R. Corey ,  Jiaxin Hu ,  Masayuki Matsui

IPC: C07H21/02 ,  C07H21/04 ,  A61K48/00 ,  C12N15/113 ,  A61K47/48

CPC classification number: C12N15/113 ,  A61K47/645 ,  C12N2310/14 ,  C12N2310/3181 ,  C12N2310/3231

Abstract: The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntington using nucleic acid analogs. Peptide nucleic acids and locked nucleic acids are particularly useful analogs.

Abstract translation: 本发明涉及使用核酸类似物选择性抑制CAG重复相关疾病蛋白如亨廷顿的蛋白质表达。 肽核酸和锁定的核酸是特别有用的类似物。