IDENTIFICATION OF ALLELE-SPECIFIC TARGET SEQUENCES FOR C9ORF72

Title:

IDENTIFICATION OF ALLELE-SPECIFIC TARGET SEQUENCES FOR C9ORF72

Document Type and Number:

WIPO Patent Application WO/2024/036186

Kind Code:

A3

Abstract:

In some embodiments, described herein are antisense oligonucleotides that target a C90rf72 sequence comprising a two-base pair deletion and G4C2 repeat expansions; and methods of using same to inhibit C90rf72 expression. In some embodiments, described herein are methods of treating amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD).

Inventors:

LANDERS JOHN EDWARD (US)
POWELL DESIREE MARIE (US)

Application Number:

PCT/US2023/071895

Publication Date:

May 16, 2024

Filing Date:

August 09, 2023

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Assignee:

UNIV MASSACHUSETTS (US)

International Classes:

C12N15/113; A61K31/7088; A61K31/7125; A61K9/00; A61P25/28

Attorney, Agent or Firm:

LECUYER, Karen A. (US)

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