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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.6K
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 in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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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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Viral Vector-Based Gene Therapy.

Xuedan Li1,2, Yang Le1,2, Zhegang Zhang1,2

  • 1National Engineering Technology Research Center for Combined Vaccines, Wuhan 430207, China.

International Journal of Molecular Sciences
|May 13, 2023
PubMed
Summary
This summary is machine-generated.

Gene therapy utilizes modified viral vectors for disease treatment, with adeno-associated viruses, adenoviruses, and lentiviruses being key strategies. Challenges like immunotoxicity and off-target effects are being addressed for improved therapeutic outcomes.

Keywords:
adeno-associated virusesadenoviruseschallengesgene therapylentiviruses

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Gene therapy offers a promising approach to treating diseases by modifying an individual's genes.
  • Efficient delivery systems are crucial for successful gene therapy.
  • Viral vectors, modified for safety, are widely used due to their high transduction efficiency and stable gene expression.

Purpose of the Study:

  • To review the three primary viral vector strategies used in gene therapy: adeno-associated viruses, adenoviruses, and lentiviruses.
  • To discuss the therapeutic applications of these viral vectors.
  • To summarize translational challenges and potential solutions in viral vector-based gene therapy.

Main Methods:

  • Review of current literature on viral vector-based gene therapy.
  • Analysis of adeno-associated viruses, adenoviruses, and lentiviruses as delivery systems.
  • Discussion of clinical outcomes, challenges, and strategies for viral vector gene therapy.

Main Results:

  • Viral vectors derived from adeno-associated viruses, adenoviruses, and lentiviruses are the leading strategies in gene therapy.
  • These vectors have demonstrated significant clinical success in various therapeutic applications.
  • Key challenges include immunotoxicity and off-target effects, necessitating further research and development.

Conclusions:

  • Adeno-associated viruses, adenoviruses, and lentiviruses are effective viral vectors for gene therapy, offering high transduction efficiency and stable expression.
  • Despite promising clinical outcomes, challenges such as immunotoxicity and off-target effects persist.
  • Ongoing research focuses on overcoming these translational hurdles to enhance the safety and efficacy of gene therapies.