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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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Related Experiment Video

Updated: Jul 1, 2025

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

Rose Kitawi1, Scott Ledger1, Anthony D Kelleher1,2,3

  • 1Kirby Institute, University of New South Wales, Kensington, NSW 2052, Australia.

International Journal of Molecular Sciences
|March 13, 2024
PubMed
Summary

Gene therapy offers hope for genetic diseases, with ex vivo methods enhancing safety and efficacy. This approach, particularly for Human Immunodeficiency Virus (HIV), allows cell selection before patient treatment, minimizing immune rejection.

Keywords:
HIVex vivogene therapystem cellsvector

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Area of Science:

  • Biotechnology
  • Genetics
  • Immunology

Background:

  • Early gene therapy efforts for heritable diseases faced setbacks due to genotoxicity, leading to cautious progress.
  • Recent advancements in genetic engineering have revived gene therapy, marked by the 2017 approval of a gene therapy product for genetic mutations.

Purpose of the Study:

  • To review the stages of ex vivo gene therapy.
  • To highlight research advancements improving ex vivo gene therapy efficiency and safety.
  • To summarize Human Immunodeficiency Virus (HIV) gene therapy studies, predominantly using ex vivo approaches.

Main Methods:

  • Ex vivo gene therapy involves modifying cells outside the body.
  • Characterization and selection of gene-modified cells prior to patient administration.
  • Utilizing autologous cells to prevent immune rejection.

Main Results:

  • Ex vivo gene therapy allows for rigorous quality control of modified cells.
  • Advancements have improved the safety and efficiency of ex vivo gene modification techniques.
  • The ex vivo approach is the predominant strategy in current Human Immunodeficiency Virus (HIV) gene therapy research.

Conclusions:

  • Ex vivo gene therapy presents a safer and more controlled method for treating genetic disorders.
  • Continued research is enhancing the efficiency and safety profile of ex vivo gene therapy.
  • The ex vivo strategy is crucial for the advancement of gene therapies, especially for complex conditions like HIV.