Endogene Intronische Rna zur Stringenten Kontrolle der Cas/crispr-Vermittelten Geneditierung in Menschlichen Zelllagen

Details

Veröffentlichungs-Nr.

EP4825970

Anmeldetag

14. April 2026

Veröffentlichung

21. Oktober 2026

Rechtsraum

EP

IPC

C12N15/113, // C12N9/22

Abstract

The present disclosure refers to a CRISPR/Cas-based gene editing system for conditional activation in human cells, characterized in that the system comprises: a Cas protein or a functionally equivalent variant thereof; and at least one conditionally activatable guide RNA (gRNA), wherein the gRNA is designed to be activated by hybridization with an endogenous intronic RNA molecule in the human cell, thereby enabling targeted DNA cleavage by the Cas protein. It further discloses an in vitro process for activating a guide RNA (gRNA) in a CRISPR/Cas-based gene editing system in human cells, characterized in that the process comprises the following steps: i) providing a conditionally activatable gRNA; ii) exposing the gRNA to an endogenous intronic RNA molecule in the human cell; iii) hybridizing the gRNA with the intronic RNA molecule, thereby activating the gRNA and enabling Cas-mediated DNA cleavage at a target genomic locus, and its use in in vitro targeted gene editing, functional genomics, and therapeutic genome engineering in human cells.

Anmelder

Firma

Egas Moniz - Cooperativa de Ensino Superior, CRL

Land

🇵🇹 Portugal

Fachgebiete