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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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Retrovirus Life Cycles01:10

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Retroviruses have a single-stranded RNA genome that undergoes a special form of replication. Once the retrovirus has entered the host cell, an enzyme called reverse transcriptase synthesizes double-stranded DNA from the retroviral RNA genome. This DNA copy of the genome is then integrated into the host’s genome inside the nucleus via an enzyme called integrase. Consequently, the retroviral genome is transcribed into RNA whenever the host’s genome is transcribed, allowing the...
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Inhibitors of Viral Protein Synthesis01:30

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Protein synthesis is indispensable for viral replication, as viruses lack the cellular machinery required for this process and must hijack the host's translational apparatus. In response, host cells deploy a critical innate immune defense involving interferons, specialized cytokines that play a central role in inhibiting viral propagation.Upon viral detection, infected cells release interferons that bind to receptors on adjacent uninfected cells, activating the JAK-STAT signaling pathway and...
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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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Updated: Apr 22, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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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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Gene therapy for HIV infection.

Carmen de Mendoza1, Pablo Barreiro, Laura Benitez

  • 1Puerta de Hierro Research Institute and University Hospital, Department of Internal Medicine , Majadahonda, Madrid , Spain cmendoza.cdm@gmail.com.

Expert Opinion on Biological Therapy
|October 18, 2014
PubMed
Summary

Gene therapy offers a potential cure for human immunodeficiency virus (HIV) by targeting infected cells and aiming to eliminate viral reservoirs. This approach seeks to overcome limitations of lifelong antiretroviral therapy and offers hope for a functional HIV cure.

Keywords:
HIVgene therapyhighly active antiretroviral therapy

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Area of Science:

  • * Gene therapy for HIV eradication.
  • * Advances in T-cell and stem cell-based strategies.

Background:

  • * Highly active antiretroviral therapy (HAART) manages HIV but requires lifelong adherence.
  • * Limitations of HAART include toxicity, cost, drug interactions, and resistance.
  • * Integrated HIV provirus cannot be eliminated, leading to viral rebound upon treatment cessation.

Purpose of the Study:

  • * To review current gene therapy strategies for HIV infection.
  • * To explore advances in HIV eradication research.
  • * To highlight the goal of eliminating latent viral reservoirs for a potential HIV cure.

Main Methods:

  • * Review of gene therapy strategies for HIV.
  • * Analysis of T-cell-based and stem cell approaches.
  • * Examination of ongoing and planned clinical trials.
  • * Literature search of PubMed and international conference proceedings.

Main Results:

  • * Gene therapy shows promise in blocking HIV infection of target cells.
  • * Engineering autologous T cells and hematopoietic stem cells resistant to HIV is under investigation.
  • * The Berlin patient serves as a proof-of-concept for HIV cure via cellular modification.

Conclusions:

  • * Gene therapy represents a revolutionary approach to achieving a functional HIV cure.
  • * Eliminating latent viral reservoirs remains the primary challenge in HIV eradication.
  • * Ongoing research aims to replicate successful HIV cure models and offers hope for ending lifelong antiretroviral therapy.