Related Experiment Video
Updated: Aug 15, 2026

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
Gene therapy for HIV-1 infection: are lethal genes a valuable tool?
1Departamento de Biología Experimental, Universidad de Jaén, Campus de las Lagunillas s/n 23071, Jaén, Spain. fjluque@ujaen.es
Cellular and Molecular Biology (Noisy-Le-Grand, France)
|September 21, 2005
Summary
Highly active antiretroviral therapy (HAART) has limitations due to persistent HIV-1 reservoirs. Gene therapy offers a potential cure by targeting and eliminating these infected cells, complementing HAART.
Area of Science:
- Virology
- Gene Therapy
- Immunology
Background:
- Highly active antiretroviral therapy (HAART) effectively suppresses HIV-1 replication but does not eliminate latent viral reservoirs.
- Persistent replication-competent HIV-1 sanctuaries compromise long-term patient health and treatment efficacy.
- Current HAART limitations necessitate novel therapeutic strategies for a functional cure.
Purpose of the Study:
- To explore gene therapy strategies for eliminating latently HIV-1 infected cells.
- To re-evaluate the potential of suicide gene therapy for HIV-1 eradication.
- To assess gene therapy as a complementary approach to HAART for a long-term HIV-1 cure.
Main Methods:
- Review of existing HIV-1 gene therapy strategies, focusing on cell killing and cell protection approaches.
- Analysis of the rationale and limitations of suicide gene therapy for targeting HIV-1 provirus expression.
- Consideration of recent advancements in lentiviral vectors for enhanced gene delivery.
Main Results:
- Suicide gene therapy, targeting infected cells for elimination, presents a viable strategy.
- Recent advancements in lentiviral vectors improve the feasibility and safety of gene therapy approaches.
- Elimination of latently infected cells is crucial for a potential HIV-1 cure.
Conclusions:
- Gene therapy, particularly suicide gene therapy, warrants reconsideration for HIV-1 treatment.
- This approach can complement HAART by targeting persistent viral reservoirs.
- Eliminating latently infected cells offers hope for a long-term cure for HIV-1 infection.
Related Concept Videos
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 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...
What is Genetic Engineering?
Overview
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...
In-vitro Mutagenesis
To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
Lethal Alleles
Agouti: A Lethal Allele
Lucien Cuénot discovered lethal alleles in 1905 while studying the inheritance of coat color in mice. The agouti gene is responsible for the color of the coat in mice. This gene codes for an agouti-signaling protein, which is responsible for melanin distribution in mammals. The wild-type allele gives rise to gray-brown coat color in mice, while the mutant allele gives rise to yellow coat color. In addition to coat color, the agouti gene is associated with the yellow...
Lucien Cuénot discovered lethal alleles in 1905 while studying the inheritance of coat color in mice. The agouti gene is responsible for the color of the coat in mice. This gene codes for an agouti-signaling protein, which is responsible for melanin distribution in mammals. The wild-type allele gives rise to gray-brown coat color in mice, while the mutant allele gives rise to yellow coat color. In addition to coat color, the agouti gene is associated with the yellow...

