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Related Concept Videos

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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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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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Prospects for Foamy Viral Vector Anti-HIV Gene Therapy.

Arun K Nalla1, Grant D Trobridge2,3

  • 1Pharmaceutical Sciences, College of Pharmacy, Washington State University Spokane, Spokane, WA 99202, USA. arun.nalla@wsu.edu.

Biomedicines
|May 25, 2017
PubMed
Summary

Foamy virus vectors show promise for human immunodeficiency virus (HIV) gene therapy by efficiently delivering anti-HIV genes into hematopoietic stem cells (HSC). This approach may overcome limitations of current retroviral vectors for HIV treatment.

Keywords:
anti-HIV transgenesfoamy virusesgene therapyretroviral vector

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Area of Science:

  • * Gene therapy for infectious diseases
  • * Hematopoietic stem cell (HSC) biology
  • * Retroviral vector technology

Background:

  • * Stem cell gene therapy is being investigated for Human Immunodeficiency Virus (HIV) infection.
  • * Existing anti-HIV gene therapies utilize retroviral vectors to deliver therapeutic genes, blocking viral replication.
  • * Gammaretroviral and lentiviral vectors face challenges in efficiently delivering anti-HIV genes into HSCs.

Purpose of the Study:

  • * To review novel anti-HIV transgenes for gene therapy.
  • * To evaluate the potential of foamy virus vectors for HIV gene therapy.
  • * To address limitations of current retroviral vectors in HSC gene delivery for HIV.

Main Methods:

  • * Review of existing clinical trials and research on stem cell gene therapy for HIV.
  • * Analysis of anti-HIV gene delivery using various retroviral vectors.
  • * Assessment of foamy virus vector characteristics for HSC gene transfer.

Main Results:

  • * Several anti-HIV genes delivered via retroviral vectors have demonstrated efficacy in blocking HIV replication.
  • * Foamy virus vectors exhibit efficient transgene delivery into HSCs, particularly in large animal models.
  • * Foamy virus vectors offer a potentially safer integration profile compared to other retroviral vectors.

Conclusions:

  • * Foamy virus vectors represent a promising alternative for HSC gene therapy in HIV treatment.
  • * Further research into novel anti-HIV transgenes and foamy virus vector optimization is warranted.
  • * This approach holds potential for overcoming current barriers in HIV gene therapy delivery.