公开(公告)号：US11840690B2

公开(公告)日：2023-12-12

申请号：US17528383

申请日：2021-11-17

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

Inventor： David Corey ,  Jiaxin Hu

IPC: C07H21/04 ,  C12N15/113 ,  A61K31/713

CPC classification number: C12N15/113 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3521 ,  C12N2320/34

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

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。

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：US10538762B2

公开(公告)日：2020-01-21

申请号：US15518824

申请日：2015-10-08

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

Inventor： David Corey ,  Jiaxin Hu

IPC: C07H21/04 ,  C12N15/113 ,  A61K31/713

Abstract: Provided herein are compositions and methods for reducing expression of C9orf72 transcripts in cells containing expanded intronic GGGGCC regions, including those in subjects having or at risk of developing amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Provided herein are a double-stranded oligonucleotides of 13 to 22 nucleobases in length targeting a GGGGCC expanded repeat region in an intron of C9orf72, comprises (a) 3-5 central mismatches (within bases 9-14) within a target sequence comprising the expanded repeat sequence, or (h) 3-5 mismatches outside of the seed sequence (bases 2-8 within the guide strand complementary to the expanded repeat sequence).