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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...

Tumor Immunotherapy

Tumor Immunotherapy

Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.

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Related Experiment Video

Updated: Feb 28, 2026

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

Genome-Edited T Cell Therapies.

Juliette M K M Delhove¹, Waseem Qasim¹

¹Molecular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London (UCL), 30 Guilford Street, London, WC1N 1EH UK.

Current Stem Cell Reports

|June 10, 2017

Summary

T cell therapies, enhanced by genome editing, show promise for cancer and viral infections. Techniques like CARs and TCRs enable precise targeting, leading to successful immunotherapies and potential for universal T cell treatments.

Keywords:

CRISPR/Cas9 Chimeric antigen receptors Clinical trials Genome editing Immunotherapy T cell receptors T cell therapies

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Last Updated: Feb 28, 2026

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

Production of Human CRISPR-Engineered CAR-T Cells

Production of Human CRISPR-Engineered CAR-T Cells

Published on: March 15, 2021

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells

Using CRISPR/Cas9 to Knock Out GM-CSF in CAR-T Cells

Published on: July 22, 2019

Area of Science:

Immunology
Molecular Biology
Biotechnology

Background:

T cell therapies offer targeted cancer treatment alternatives to conventional drugs.
Recombinant T cell receptors (TCRs) and chimeric antigen receptors (CARs) enable specific cell targeting.
Genome editing tools are crucial for advancing T cell therapy development.

Purpose of the Study:

Review genome editing tools for T cell therapies.
Highlight site-specific DNA nuclease applications.
Discuss TCR and CAR T cell therapy advancements.

Main Methods:

Review of literature on T cell therapies and genome editing.
Focus on site-specific DNA nucleases (e.g., CRISPR-Cas9).
Analysis of TCR and CAR engineering techniques.

Main Results:

TCR and CAR engineering have led to successful immunotherapies.
Genome editing enables HLA-independent universal T cells for transplantation.
PD-1 knockout improves T cell sustainability; antiviral efficacy demonstrated.

Conclusions:

Combined engineered antigen-targeting and genome editing show success in clinical trials for HIV and hematological malignancies.
These innovative strategies are being integrated into broader immunotherapy approaches.
Future applications include expanding T cell therapy to other indications.