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COMPOSITIONS AND METHODS FOR REDUCING RNA LEVELS

Application US20260085311A1 Kind: A1 Mar 26, 2026

Inventors

Liana Lareau, Fyodor Urnov, Ma. Carmelle Catamura

Abstract

The present disclosure provides methods for reducing the level of an RNA transcript from a target nucleic acid. The present disclosure provides methods of treating a disease that results from or is caused by a toxic gain-of-function protein. The present disclosure provides systems and compositions for carrying out a method of the present disclosure.

CPC Classifications

C12N 15/113 A61K 38/00 A61K 48/00 C12N 9/1276 C12N 9/222 C12N 9/78 C12N 15/52 C12N 15/63 C12Y 207/07049 C12Y 305/04004 C12Y 305/04005 C12N 2310/20

Filing Date

2023-06-20

Application No.

18876098