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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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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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Related Experiment Video

Updated: May 1, 2026

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 targeting HIV entry.

Chuka Didigu1, Robert Doms2

  • 1Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, 3610 Hamilton Walk, Philadelphia, PA 19104, USA. cdidigu@mail.med.upenn.edu.

Viruses
|March 26, 2014
PubMed
Summary
This summary is machine-generated.

Developing a cure for HIV is crucial due to treatment limitations. Allogeneic stem cell transplants and gene therapy show promise for achieving an HIV cure, offering new research avenues.

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Area of Science:

  • Immunology
  • Infectious Diseases
  • Gene Therapy

Background:

  • Antiretroviral therapy (ART) controls HIV but doesn't cure it.
  • Persistent morbidities and adherence challenges necessitate a definitive HIV cure.
  • Recent advances in stem cell transplantation offer potential pathways to cure HIV.

Purpose of the Study:

  • To review current obstacles in HIV cure research.
  • To explore the potential of gene therapy and stem cell transplantation for HIV cure.
  • To highlight emerging strategies for eradicating HIV infection.

Main Methods:

  • Review of current scientific literature on HIV cure strategies.
  • Analysis of allogeneic hematopoietic stem cell transplantation (HSCT) outcomes.
  • Evaluation of gene therapy approaches targeting HIV entry.

Main Results:

  • Allogeneic HSCT has demonstrated the potential for HIV cure, even with susceptible cells.
  • Gene therapy offers a promising strategy to block HIV entry.
  • Significant obstacles remain in the development of a widely applicable HIV cure.

Conclusions:

  • A functional HIV cure may be achievable through advanced therapeutic strategies.
  • Allogeneic HSCT and gene therapy are key areas of investigation for HIV eradication.
  • Continued research is vital to overcome challenges and develop effective HIV cures.