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Related Experiment Video

Updated: Jan 9, 2026

Generation of Human Chimeric Antigen Receptor Regulatory T Cells

Generation of Human Chimeric Antigen Receptor Regulatory T Cells

Published on: January 3, 2025

Gene-corrected regulatory T cell therapy for IL2RA deficiency.

Alvin S Ha¹, Nechama Kalter², Michael Rosenberg²

¹Department of Laboratory Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Gladstone-UCSF Institute of Genomic Immunology, San Francisco, CA 94158, USA.

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

|December 11, 2025

Summary

This summary is machine-generated.

Gene-corrected regulatory T cells (Tregs) offer a potential new therapy for IL2RA deficiency, a rare autoimmune disease. This approach uses CRISPR-Cas9 to fix gene mutations, creating functional Tregs for a safer treatment than current options.

Keywords:

CRISPR IL2RA clinical manufacturing gene correction inborn errors of immunity non-viral rare disease regulatory T cells

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Last Updated: Jan 9, 2026

Generation of Human Chimeric Antigen Receptor Regulatory T Cells

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Published on: January 3, 2025

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

Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity

Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity

Published on: November 8, 2016

Area of Science:

Immunology
Gene Therapy
Cell Therapy

Background:

Bi-allelic germline deficiency of IL2RA causes a rare autoimmune condition characterized by impaired regulatory T cell (Treg) function and interleukin-2 (IL-2) signaling.
Current definitive treatment, allogeneic hematopoietic stem cell transplantation, involves significant risks.

Purpose of the Study:

To develop a novel therapeutic approach for IL2RA deficiency using ex vivo gene-corrected autologous Tregs (gcTregs).
To establish a Good Manufacturing Practice (GMP)-compatible manufacturing process for personalized gcTreg therapies.

Main Methods:

CRISPR-Cas9-mediated homology-directed repair was used to correct one of two disease-causing mutations in patient-derived Tregs.
Gene correction aimed to restore IL2RA expression and Treg function.
Clinical-scale manufacturing processes were developed and validated.

Main Results:

The gcTregs demonstrated restored IL2RA expression and robust in vitro suppressive activity.
Clinical-scale manufacturing achieved efficient gene correction and functional equivalence to healthy donor Tregs.
The process was shown to be GMP-compatible.

Conclusions:

Personalized gcTreg therapy represents a potentially safer treatment for IL2RA deficiency compared to current options.
This approach provides a framework for treating other inborn errors of immunity involving Treg dysfunction.