Chemically modified guide rnas for crisper / cas-mediated gene regulation
CN114231527BActive Publication Date: 2026-06-19THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV +1
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Patents(China)
- Current Assignee / Owner
- THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV
- Filing Date
- 2016-04-05
- Publication Date
- 2026-06-19
AI Technical Summary
Technical Problem
Existing CRISPR/Cas systems are less efficient at genome editing and gene expression regulation in primary cells, especially in in vitro and in vivo therapies.
Method used
Using chemically modified single guide RNA (sgRNA) containing a first nucleotide sequence complementary to the target nucleic acid and a second nucleotide sequence that interacts with a CRISPR-associated protein (Cas) peptide, the gene editing and gene expression regulation of the target nucleic acid are enhanced by binding to the Cas peptide.
Benefits of technology
It significantly improved the efficiency and specificity of gene editing in primary cells, effectively corrected mutations related to genetic diseases, and enhanced the accuracy and stability of genome editing.
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Figure CN114231527B_ABST
Abstract
This article provides a method for inducing CRISPR / Cas-based gene regulation (e.g., genome editing or gene expression) of target nucleic acids (e.g., target DNA or target RNA) in cells. The method includes using a modified single guide RNA (sgRNA) that enhances gene regulation of the target nucleic acid in primary cells used for in vitro therapy or in subject cells used for in vitro therapy. Additionally, this article provides a method for preventing or treating genetic diseases in subjects by administering sufficient amounts of modified sgRNA to correct mutations in target genes associated with genetic diseases.
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