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Related Concept Videos

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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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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: Jun 5, 2025

Immunometabolic Circuits in Infection for Advancing Host Directed Therapies
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Immunometabolic Circuits in Infection for Advancing Host Directed Therapies

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Synthetic gene circuits drive disease-fighting T cells.

Marco L Davila1, Renier Brentjens1

  • 1Roswell Park Comprehensive Cancer Center, Elm and Carlton Streets, Buffalo, NY, USA.

Science (New York, N.Y.)
|December 5, 2024
PubMed
Summary

Immune cells are being engineered to deliver targeted therapies for brain and inflammatory conditions. This approach shows promise for treating complex diseases by directing therapeutic agents precisely where needed.

Area of Science:

  • Immunology
  • Neuroscience
  • Pharmacology

Background:

  • Targeted therapy delivery remains a challenge in treating brain and inflammatory diseases.
  • Conventional treatments often lack specificity, leading to off-target effects and limited efficacy.

Purpose of the Study:

  • To investigate the potential of programming immune cells for targeted therapeutic delivery.
  • To evaluate the efficacy of engineered immune cells in preclinical models of neurological and inflammatory disorders.

Main Methods:

  • Utilizing genetic engineering techniques to modify immune cells.
  • Developing and testing these engineered cells in in vitro and in vivo disease models.
  • Assessing the biodistribution and therapeutic effects of the programmed immune cells.

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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
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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

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Main Results:

  • Demonstrated successful programming of immune cells for targeted homing to disease sites.
  • Observed significant therapeutic benefits in models of brain and inflammatory conditions.
  • Confirmed the safety and specificity of the engineered immune cell-based therapy.

Conclusions:

  • Engineered immune cells represent a viable strategy for targeted therapy delivery in neurological and inflammatory diseases.
  • This innovative approach holds potential for developing more effective and safer treatment options.