公开(公告)号：US11795457B2

公开(公告)日：2023-10-24

申请号：US17486136

申请日：2021-09-27

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

Inventor： David Corey ,  Liande Li

IPC: C07H21/04 ,  C12N15/113 ,  A61P25/00 ,  A61K45/06

CPC classification number: C12N15/113 ,  A61K45/06 ,  A61P25/00 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/3231

Abstract: Described are compounds and methods useful for the treatment and investigation of Friedreich's Ataxia.

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：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).