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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

Precision T cell correction platform for inborn errors of immunity.

Katariina Mamia¹, Solrun Kolbeinsdottir², Kornel Labun³

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

Molecular Therapy : the Journal of the American Society of Gene Therapy

|August 14, 2025

Summary

This summary is machine-generated.

This study introduces a precise CRISPR/Cas9 gene editing tool for correcting single gene defects in T cells, offering a new therapy for Inborn errors of immunity (IEI) without harmful side effects.

Keywords:

CRISPR-Cas9 gene correction autologous T cell therapy ex vivo gene editing gene therapy homology-directed repair inborn errors of immunity non-viral genome editing platform technology primary T cell editing single-nucleotide variant correction

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

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

Published on: February 28, 2025

Area of Science:

Immunology
Gene Therapy
Molecular Biology

Background:

Current gene editing strategies for Inborn errors of immunity (IEI) can disrupt gene expression.
Autologous hematopoietic stem cell transplantation is not suitable for all patients.
T cell gene correction offers an alternative therapeutic strategy for lymphoid IEIs.

Purpose of the Study:

To develop and validate an efficient T cell single nucleotide variant (SNV) correction platform using homology-directed repair (HDR).
To assess the efficacy and safety of the HDR-based platform in preclinical models of IEI.

Main Methods:

Utilized homology-directed repair (HDR) for precise SNV correction in T cells.
Employed IEI models including STAT1 gain-of-function (STAT1-GOF), Cartilage Hair Hypoplasia (CHH), Deficiency of ADA2 (DADA2), and Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED).
Conducted comprehensive safety profiling using GUIDE-seq, single-cell RNA sequencing, long-read genome sequencing, and proteomics.

Main Results:

Achieved up to 80% correction efficiency in the selected IEI models.
Demonstrated functional correction of disease phenotypes in preclinical models.
Confirmed no significant genomic, transcriptomic, or proteomic aberrations post-editing.

Conclusions:

Established HDR-based SNV editing as a viable and portable platform for autologous T cell therapies.
This approach represents a significant advancement toward a broad-spectrum gene correction strategy for monogenic immune disorders.
The platform shows promise for clinical development in treating diverse IEIs.