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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers
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Bringing gene therapy to where it's needed.

Stefan Radtke1, Hans-Peter Kiem2

  • 1Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA.

Trends in Molecular Medicine
|January 28, 2022
PubMed
Summary

Virus-like particles (VLPs) offer a novel method for delivering gene-editing tools directly within the body. This breakthrough overcomes a major hurdle in gene therapy, enabling precise genetic modifications for therapeutic applications.

Keywords:
base editorsgene therapyin vivo deliverytarget specificity

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Gene Therapy

Background:

  • Gene editing technologies enable precise cellular modifications for treating genetic disorders and improving immunotherapies.
  • A significant challenge in gene therapy is the efficient and targeted delivery of gene-editing components to specific cells or organs.
  • Current delivery methods often face limitations in specificity and in vivo application.

Purpose of the Study:

  • To introduce and evaluate virus-like particles (VLPs) as a novel delivery system for gene-editing agents.
  • To demonstrate the potential of VLPs for in vivo gene therapy applications.
  • To overcome the delivery limitations associated with current gene-editing technologies.

Main Methods:

  • Development of virus-like particles (VLPs) engineered to encapsulate gene-editing agents.
  • In vivo administration of VLP-loaded gene-editing tools.
  • Assessment of VLP-mediated delivery and gene-editing efficiency in target cells or tissues.

Main Results:

  • Successful loading of gene-editing agents within virus-like particles (VLPs).
  • Demonstration of VLP-mediated delivery of gene-editing tools directly inside the body.
  • Evidence of precise genetic modification in target cells following VLP administration.

Conclusions:

  • Virus-like particles (VLPs) represent a promising platform for in vivo gene therapy delivery.
  • This VLP-based approach effectively addresses the challenge of delivering gene-editing technologies to specific sites within the body.
  • The findings pave the way for enhanced gene-editing therapies with improved targeting and efficacy.