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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

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Related Experiment Video

Updated: Jun 26, 2025

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

Targeted gene delivery systems for T-cell engineering.

Fengling Wang¹, Yong Huang¹, JiaQian Li¹

¹Department of Biotherapy, State Key Laboratory of Biotherapy and Cancer Center, West China Hospital, Sichuan University, Chengdu, 610041, People's Republic of China.

Cellular Oncology (Dordrecht, Netherlands)

|May 16, 2024

Summary

Harnessing T lymphocytes for therapies requires advanced gene delivery. This review explores viral and nonviral vectors, focusing on in vivo T-cell modification for improved immunotherapies.

Keywords:

Nonviral vector T-cell based immunotherapy Targeted gene delivery system Viral vector

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Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

Published on: February 1, 2013

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

Published on: October 6, 2023

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Last Updated: Jun 26, 2025

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

Published on: February 1, 2013

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

Published on: October 6, 2023

Area of Science:

Immunology
Biotechnology
Gene Therapy

Background:

T lymphocytes are crucial for host defense against pathogens and tumors.
Genetically engineered T-cells, like CAR-T and TCR-T, show promise in treating cancer and autoimmune diseases.
Current T-cell engineering relies on complex in vitro isolation and modification, delaying therapy development.

Purpose of the Study:

To review gene delivery systems for T cells, including viral and nonviral vectors.
To assess the potential of in vivo T-cell gene modification strategies.
To provide insights into advancing T-cell based immunotherapies.

Main Methods:

Analysis of viral gene delivery vectors for T cells.
Evaluation of nonviral gene delivery vectors for T cells.
Review of current research on in vivo T-cell gene modification systems.

Main Results:

Viral vectors offer high transduction efficiency but face immunogenicity and production challenges.
Nonviral vectors are safer and scalable but often have lower transduction efficiency.
Targeted in vivo gene transfer to T cells without isolation is a significant advancement.

Conclusions:

Developing efficient in vivo gene delivery systems for T cells is critical for future immunotherapies.
Overcoming the limitations of current viral and nonviral vectors is essential.
In vivo T-cell modification holds revolutionary potential for treating diseases.