US 2022/0009968 A1 · CRISPR-CAS-BASED COMPOSITION FOR GENE CORRECTION

Abstract

The present disclosure relates to a composition for enhancing the cell permeability and gene correction efficiency of Cas protein and guide RNA. The currently used CRISPR-Cas-based gene correction technology has the problems of difficult intracellular injection in a complex form, unverified stability and low efficiency even after injection, and the off-target problem. In contrast, the composition for gene correction of the present disclosure can be usefully used for gene therapy due to remarkably high intracellular delivery efficiency, inhibited off-target, and ensured stability.

Claims13

Dependent claims

A cell-penetrating peptide for a Cas protein-RNA complex (ribonucleoprotein:

RNP), represented by General Formula 1:
Arg-Arg-Arg-Trp-Cys-Lys-Arg-Arg-Arg-Ala-Ser-[Gly]m[His-Glu]n [General Formula 1]
wherein m is an integer from 3 to 7, and n is an integer from 5 to 15.

Dependencies are inferred from the claim text.

Facts

Application

17/312,174Filed 27 Dec 2019

Priority

KR 10-2018-017046327 Dec 2018

Published

13 Jan 2022

Term ends

Not estimatedThe source records don’t support an estimate.

Classification12 codes

C07K 14/00
Peptides having more than 20 amino acids
C12N 15/90
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation: stable introduction of foreign DNA…
C12N 9/22
Acting on ester bonds: ribonucleases [RNase]; Deoxyribonucleases [DNase]
C12N 9/226
Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes: class 2 CAS enzyme complex, e.g. single CAS protein

Crispr-cas-based composition for gene correction

Life of this patent

Crispr-cas-based composition for gene correction

Life of this patent

Abstract

Claims13