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Related Concept Videos

Gene Therapy00:59

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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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Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
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Gene delivery for immunoengineering.

Sarah Y Neshat1, Stephany Y Tzeng1, Jordan J Green2

  • 1Department of Biomedical Engineering and the Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA.

Current Opinion in Biotechnology
|June 20, 2020
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Summary
This summary is machine-generated.

Gene delivery strategies are advancing immunoengineering for diseases like cancer. Both viral and non-viral methods are explored for effective immune cell modulation and therapeutic potential in clinical trials.

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Area of Science:

  • Biotechnology
  • Immunology
  • Gene Therapy

Background:

  • Gene delivery is crucial for immunoengineering applications, including infectious disease prevention and cancer therapy.
  • Viral vectors offer high gene transfer but face immunogenicity challenges.
  • Non-viral methods, such as nanoparticles and physical techniques, are actively researched alternatives.

Purpose of the Study:

  • To review current gene delivery strategies for immunoengineering.
  • To highlight methods for modulating immune responses through genetic programming.
  • To discuss the therapeutic potential and clinical progression of these biotechnologies.

Main Methods:

  • Utilizing viral vectors for high gene transfer capability.
  • Employing non-viral methods like polymeric, lipid-based, and inorganic nanoparticles.
  • Implementing physical delivery techniques for gene transfer.
  • Applying ex vivo immune cell engineering and in vivo cell transfection.
  • Delivering plasmid DNA, mRNA, oligonucleotides, and immunostimulatory nucleic acids.

Main Results:

  • Development of precise immune response modulation through various gene delivery approaches.
  • Successful intracellular delivery of genetic material (DNA, mRNA) for gene expression.
  • Application of oligonucleotides for gene knockdown and nucleic acids for immune tuning.
  • Advancement of numerous gene delivery biotechnologies into clinical trials.

Conclusions:

  • Gene delivery strategies are pivotal in immunoengineering with broad therapeutic applications.
  • A diverse toolkit of viral and non-viral methods allows for tailored immune modulation.
  • Ongoing clinical trials indicate significant future impact on medicine.