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Gene Therapy

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

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 Therapeutics

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 Cycles

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...

Human Virome

Human Virome

The human body harbors a vast and diverse viral community known as the human virome. The virome includes bacteriophages that infect bacteria, and eukaryotic viruses that infect human cells. Transient dietary and environmental viruses also contribute to this dynamic ecosystem. Estimates suggest the human body may contain on the order of 10¹³ viral particles, though abundance varies widely by body site and detection method.Comprehensive characterization of the virome has become possible only with...

Size and Structure of Viral Genomes

Size and Structure of Viral Genomes

Viral genomes exhibit remarkable diversity in size, structure, and composition, influencing their replication strategies and interactions with host cells. These genomes consist of either DNA or RNA and may be linear or circular. Additionally, they can be single-stranded or double-stranded, with each configuration affecting how the virus propagates within a host. RNA viruses, for instance, generally have smaller genomes than DNA viruses, a factor that contributes to their high mutation rates and...

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Related Experiment Video

Updated: May 13, 2026

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

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

Published on: September 5, 2016

HIV gene therapy research advances.

Jeffrey M Jacobson¹

¹Drexel University College of Medicine.

|March 2, 2013

Summary

This study explored antiviral effects of lentiviral vector-modified T-lymphocytes in HIV patients. The treatment showed promise in managing the virus in individuals on antiretroviral therapy.

Area of Science:

Immunotherapy
Virology
Gene Therapy

Background:

Human Immunodeficiency Virus (HIV) remains a global health challenge.
Antiretroviral therapy (ART) controls viral replication but does not eliminate the virus.
Novel therapeutic strategies are needed for HIV cure.

Purpose of the Study:

To evaluate the antiviral effects of lentiviral vector-modified autologous CD4+ T lymphocytes.
To assess the safety and efficacy of this gene therapy approach in HIV-infected patients.

Main Methods:

A clinical trial involving 17 HIV-infected patients was conducted.
Patients received multiple infusions of autologous CD4+ T lymphocytes genetically modified using a lentiviral vector.
All patients were aviremic on stable antiretroviral therapy (ART) at the time of the trial.

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Oral Combinational Antiretroviral Treatment in HIV-1 Infected Humanized Mice

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Interview: HIV-1 Proviral DNA Excision Using an Evolved Recombinase

Published on: June 16, 2008

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Last Updated: May 13, 2026

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

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

Published on: September 5, 2016

Oral Combinational Antiretroviral Treatment in HIV-1 Infected Humanized Mice

Oral Combinational Antiretroviral Treatment in HIV-1 Infected Humanized Mice

Published on: October 6, 2022

Interview: HIV-1 Proviral DNA Excision Using an Evolved Recombinase

Interview: HIV-1 Proviral DNA Excision Using an Evolved Recombinase

Published on: June 16, 2008

Main Results:

The study reported observed antiviral effects following the infusions.
Further details on viral load, CD4+ T cell counts, and immune responses were assessed.

Conclusions:

Lentiviral vector-mediated gene therapy holds potential as a novel treatment for HIV.
The findings suggest a possible role for modified T-lymphocytes in controlling HIV infection.