Method for gene editing of HLA-DRA locus

By designing sgRNA and Cas9 nuclease that specifically recognize the HLA-DRA gene, the HLA-DRA gene is efficiently knocked out, solving the problems of high off-target risk and low efficiency in existing technologies, and improving the safety and efficacy of universal CAR-T cells.

WO2025223090A1 Publication Date: 2025-10-30NANJING MIRACLE BIOTECHNOLOGY CO LTD
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Patent Information

Application Number
PCT/CN2025/082119
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-04-23
Filing Date
2025-03-12
Publication Date
2025-10-30

AI Technical Summary

Technical Problem

Existing sgRNAs have a high risk of off-target effects and low efficiency when knocking out the HLA-DRA gene, leading to frequent immune rejection reactions in allogeneic CAR-T cell therapy and limiting their clinical application.

Method used

We designed sgRNAs that specifically recognize the target site of the HLA-DRA gene, combined with the Cas9 nuclease, and efficiently knocked out the HLA-DRA gene using the CRISPR/Cas9 system to reduce cellular immunogenicity and prepare universal CAR-T cells.

Benefits of technology

This method achieves efficient, stable, and specific knockout of the HLA-DRA gene, significantly reducing the risk of host anti-graft reaction and immune rejection, and improving the safety and broad applicability of CAR-T cell therapy.

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Abstract

Provided are an sgRNA that targets and directs a nuclease to efficiently cleave an HLA-DRA gene, a method for modifying CAR-T cells using the sgRNA, and related gene editing system, agent, and kit. The provided CAR-T cells significantly reduce immunogenicity, which effectively decreases the risks of graft-versus-host disease and immune rejection, and also maintaining the killing activity thereof against tumor cells.
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