Patent · US Active

Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat

US10538762B2 · kind B2 · utility

0Cited by

2References

22Claims

0Family size

Assignee

The Board of Regents of the University of Texas System · US

Inventors

David R. Corey · Dallas, US
Jiaxin Hu · Coppell, US

Key dates

Filing date	Oct 8, 2015
Grant date	Jan 21, 2020
Priority date	—
Expiry date	Oct 8, 2035

Classification

Technology area (CPC C)Chemistry; Metallurgy
CPC primaryC12N2320/34
WIPO fieldPharmaceuticals
WIPO sectorChemistry

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).

Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.