US 2023/0416725 A1 · SYSTEMS FOR GENE EDITING AND METHODS OF USE THEREOF

Abstract

This disclosure provides a novel gene editing method and system, termed dasCRISPR. dasCRISPR refines current approaches in gene editing by allowing an intended modification to be made in one genomic target while preventing modification to a second genomic target through protective sequestration. This balancing of repair and protection contributes to cellular survival by retention of one functional copy of a gene. Thus, dasCRISPR method as disclosed allows successful CRISPR gene editing of an intended modification at a target sequence by an active Cas polypeptide, while preserving specific genomic regions, protected by dCas polypeptide, from unintended modification

Claims42

Dependent claims

A system for gene editing, comprising:

a CRISPR-associated protein (Cas) polypeptide or a first Cas nucleotide sequence encoding a Cas polypeptide;
a nuclease-deficient Cas (dCas) polypeptide or a second Cas nucleotide sequence encoding a dCas polypeptide;
and
a guide nucleotide sequence encoding or comprising a crRNA sequence capable of hybridizing with a first target sequence on a first allele and a second target sequence on a second allele and forming a complex with the Cas polypeptide and the dCas polypeptide,
wherein the Cas polypeptide binds to the first target sequence on the first allele and induces genetic modification in the first target sequence, and
wherein the dCas polypeptide binds to the second target sequence on the second allele and protects the second target sequence from modification and from the activity of the Cas polypeptide

3 more elements

Dependencies are inferred from the claim text.

Systems for gene editing and methods of use thereof

Life of this patent

Systems for gene editing and methods of use thereof

Life of this patent

Abstract

Claims42