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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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.
Retrovirus Life Cycles01:10

Retrovirus Life Cycles

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 retrovirus to...

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Related Experiment Video

Updated: May 20, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
12:03

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

Published on: September 5, 2016

Current progress and challenges in HIV gene therapy.

Janet Chung1, John J Rossi, Ulrike Jung

  • 1Division of Molecular & Cell Biology, Beckman Research Institute of the City of Hope, 1500 East Duarte Road, CA 91010, USA.

Future Virology
|July 4, 2012
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a potential cure for Human Immunodeficiency Virus type 1 (HIV-1) by creating resistant immune cells. This approach, inspired by a functional cure, aims to overcome limitations of current treatments and eradicate the virus.

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Last Updated: May 20, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Published on: September 5, 2016

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11:16

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Published on: February 15, 2019

Area of Science:

  • * Infectious Diseases
  • * Immunology
  • * Gene Therapy

Background:

  • * Human Immunodeficiency Virus type 1 (HIV-1) infection leads to Acquired Immunodeficiency Syndrome (AIDS), impacting millions worldwide.
  • * Current Highly Active Antiretroviral Therapy (HAART) manages disease progression but fails to eradicate the virus, with side effects and drug resistance posing significant challenges.
  • * The emergence of drug-resistant HIV-1 strains necessitates the development of novel therapeutic strategies.

Purpose of the Study:

  • * To review the current advancements and potential of gene therapy as a curative approach for HIV-1 infection.
  • * To highlight the challenges and obstacles hindering the widespread clinical application of HIV-1 gene therapy.

Main Methods:

  • * Review of existing literature on HIV-1 gene therapy strategies.
  • * Analysis of case studies, including the 'Berlin patient's' functional cure.
  • * Discussion of immunological and virological aspects relevant to gene therapy efficacy.

Main Results:

  • * Gene therapy can reconstitute the immune system with cells resistant to HIV-1, offering a potential pathway to a functional cure.
  • * The 'Berlin patient' case demonstrated the feasibility of achieving an undetectable HIV-1 viral load through gene therapy targeting the CCR5 receptor.
  • * Successful gene therapy can block viral entry and provide long-term control of HIV-1 infection.

Conclusions:

  • * Gene therapy represents a promising alternative to conventional treatments for HIV-1, with the potential for a functional cure.
  • * Overcoming challenges related to delivery, safety, and long-term efficacy is crucial for the clinical success of HIV gene therapy.
  • * Continued research and development are essential to translate the potential of gene therapy into a widely accessible HIV-1 cure.