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Related Concept Videos

Tumor Immunotherapy01:27

Tumor Immunotherapy

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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: Jan 16, 2026

In Vitro Tumor Cell Rechallenge For Predictive Evaluation of Chimeric Antigen Receptor T Cell Antitumor Function
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In vivo chimeric antigen receptor (CAR)-T cell therapy.

Adrian Bot1, Andrew Scharenberg2, Kevin Friedman3

  • 1Capstan Therapeutics, San Diego, CA, USA. abot@capstantx.com.

Nature Reviews. Drug Discovery
|September 30, 2025
PubMed
Summary
This summary is machine-generated.

In vivo CAR-T cell engineering generates CAR-T cells inside the body, overcoming manufacturing limits of traditional therapies. This approach shows promise for treating blood cancers and autoimmune diseases.

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Area of Science:

  • Immunotherapy
  • Cellular Therapy
  • Genetic Engineering

Background:

  • Chimeric antigen receptor (CAR)-T cell therapy has revolutionized hematological cancer treatment.
  • Current ex vivo CAR-T cell therapy faces challenges in manufacturing, scalability, and clinical consistency.

Purpose of the Study:

  • To review advances in in vivo CAR-T cell engineering.
  • To explore technologies enabling in vivo CAR-T cell generation.
  • To discuss the potential of in vivo approaches for broader therapeutic applications.

Main Methods:

  • Utilizing targeted delivery systems like lentiviral vectors and lipid nanoparticles.
  • Introducing CAR-encoding genetic material into endogenous T cells within the patient.
  • Leveraging advances in virology, RNA medicines, and nanotechnology.

Main Results:

  • Early clinical studies demonstrate efficient T cell transduction and sustained CAR expression.
  • Initial evidence of antitumour activity establishes proof of concept for in vivo CAR-T generation.
  • In vivo engineering eliminates the need for ex vivo cell processing and conditioning.

Conclusions:

  • In vivo CAR-T cell engineering offers a scalable and potentially more effective alternative to ex vivo methods.
  • This strategy could expand CAR-T cell therapy applications beyond hematological malignancies to autoimmune diseases.
  • Technological advancements are driving the development of safer and more broadly applicable in vivo CAR-T cell therapies.