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Updated: Jul 1, 2026

Generation of Induced Regulatory T Cells from Primary Human Naïve and Memory T Cells

Generation of Induced Regulatory T Cells from Primary Human Naïve and Memory T Cells

Published on: April 16, 2012

针对免疫的先天性错误的精密T细胞校正平台.

Katariina Mamia¹, Solrun Kolbeinsdottir², Kornel Labun³

¹Centre for Molecular Medicine Norway, University of Oslo; Oslo, 0318, Norway; Department of Pediatrics, Oslo University Hospital; Oslo, 0372, Norway; Precision Immunotherapy Alliance, University of Oslo; Oslo, 0379, Norway.

Molecular therapy : the journal of the American Society of Gene Therapy

|August 14, 2025

概括

这项研究引入了一种精确的CRISPR/Cas9基因编辑工具,用于纠正T细胞中的单基因缺陷,为免疫的先天性错误 (IEI) 提供了一种新的治疗方法,没有有害的副作用.

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Electroporation-Based CRISPR-Cas9-Mediated Gene Knockout in THP-1 Cells and Single-Cell Clone Isolation

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Last Updated: Jul 1, 2026

Generation of Induced Regulatory T Cells from Primary Human Naïve and Memory T Cells

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Published on: April 16, 2012

Genome Editing and Directed Differentiation of hPSCs for Interrogating Lineage Determinants in Human Pancreatic Development

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Electroporation-Based CRISPR-Cas9-Mediated Gene Knockout in THP-1 Cells and Single-Cell Clone Isolation

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科学领域:

免疫学免疫学免疫学
基因治疗基因治疗
分子生物学分子生物学

背景情况:

目前用于免疫的先天性错误 (IEI) 的基因编辑策略可以破坏基因表达.
自主造血干细胞移植不适合所有患者.
T细胞基因校正为淋巴细胞IEI提供了替代治疗策略.

研究的目的:

开发和验证一个高效的T细胞单核酸变异 (SNV) 校正平台,使用同质导向修复 (HDR).
在IEI的临床前模型中评估基于HDR的平台的有效性和安全性.

主要方法:

使用同质导向修复 (HDR) 来精确纠正T细胞中的SNV.
采用了IEI模型,包括STAT1功能增益 (STAT1-GOF),软骨头发低质化 (CHH),ADA2缺乏 (DADA2) 和自身免疫多样性肌内膜异位症 - 候群病 - 皮肤外 (APECED).
使用GUIDE-seq,单细胞RNA测序,长读基因组测序和蛋白质组学进行了全面的安全分析.

主要成果:

在选定的IEI模型中达到高达80%的校正效率.
在临床前模型中证明了疾病表型的功能纠正.
经过编辑后确认没有显著的基因组,转录组或蛋白质组异常.

结论:

建立了基于HDR的SNV编辑,作为自主T细胞疗法的可行和便携平台.
这种方法代表了向单一性免疫疾病的宽谱基因校正策略的重大进步.
该平台对治疗各种IEI的临床开发充满希望.