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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 inserted. The...

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 inserted. The...

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: May 24, 2026

A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy

A Nonviral Approach to Generate Transient Chimeric Antigen Receptor T Cells Using mRNA for Cancer Immunotherapy

Published on: February 21, 2025

In vivo gene therapy with CAR-T cells.

Dimitrios L Wagner¹, Magdi Elsallab², Marcela V Maus³

¹Baylor College of Medicine, Houston, Texas, United States.

|May 22, 2026

Summary

This summary is machine-generated.

In vivo generation of chimeric antigen receptor (CAR) T cells offers a promising alternative to complex ex vivo manufacturing for treating blood cancers. This approach shows therapeutic potential, with ongoing research addressing key technical and safety aspects.

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Area of Science:

Immunology
Gene Therapy
Oncology

Background:

Ex vivo manufacturing of chimeric antigen receptor (CAR) T cells is effective for lymphoid malignancies but logistically challenging.
In vivo generation of CAR T cells presents an alternative approach with potential benefits and unique challenges.

Purpose of the Study:

To review clinical evidence supporting in vivo CAR gene delivery.
To discuss critical technical, biological, and safety considerations for in vivo CAR T cell generation.

Main Methods:

Literature review of recent clinical reports on in vivo CAR gene delivery.
Analysis of technical, biological, and safety data from clinical studies.

Main Results:

Clinical reports demonstrate the feasibility and therapeutic potential of in vivo CAR gene delivery.
Key considerations for successful in vivo CAR T cell therapy are identified.

Conclusions:

In vivo CAR gene delivery is a viable strategy with significant therapeutic promise.
Addressing technical, biological, and safety challenges is crucial for advancing this approach in cancer treatment.