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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...

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...

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.

Retroviruses

Retroviruses

Retroviruses and retrotransposons both insert copies of their genetic elements into the genome of the host cell. Thus, the viral genes are passed on when the host genome is replicated or translated. A typical retroviral DNA sequence contains 3-4 genes that encode the different proteins required for its structural assembly and function as a molecular parasite. This DNA is transcribed into a single mRNA, which is very similar in structure to conventional mRNAs, i.e., it is capped at the 5’...

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Updated: Nov 24, 2025

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: An Update.

Tatjana I Cornu^1,2,3, Claudio Mussolino^1,2,3, Matthias C Müller^3,4,5

¹Institute for Transfusion Medicine and Gene Therapy, Medical Center-University of Freiburg, Freiburg, Germany.

Human Gene Therapy

|December 22, 2020

Summary

This summary is machine-generated.

Cell and gene therapies show promise for treating HIV, aiming for a functional cure. Current approaches face challenges, but ongoing research explores innovative one-time treatments with minimal toxicity for HIV patients.

Keywords:

AIDS CCR5 CRISPR-Cas epigenome editing gene editing latent HIV reservoir

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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

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

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Published on: June 16, 2008

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Published on: October 6, 2022

Area of Science:

Immunology
Virology
Biotechnology

Background:

Antiretroviral therapy has improved HIV/AIDS patient quality of life but faces challenges like drug resistance and lifelong treatment needs.
Developing an effective HIV vaccine remains elusive, prompting research into alternative therapeutic strategies.

Purpose of the Study:

To review clinical and technical challenges in HIV treatment.
To summarize promising cell and gene therapy concepts for a functional HIV cure.
To discuss socioeconomic factors and future directions for HIV cell and gene therapies.

Main Methods:

Review of current scientific literature on HIV cell and gene therapies.
Analysis of clinical and technical challenges in HIV treatment.
Exploration of novel therapeutic strategies including CAR T-cell therapy, gene therapy, and genome editing.

Main Results:

Despite progress, no cell or gene therapy has yet achieved a functional cure for HIV.
Innovative approaches like CAR T-cells, gene therapy, and genome editing show potential but require further development.
Challenges include treatment toxicity, adherence, and the need for lifelong therapy.

Conclusions:

Cell and gene therapies offer potential for a one-time, low-toxicity treatment for HIV.
Further research and development are crucial to overcome existing challenges and achieve a functional cure.
Addressing socioeconomic aspects is vital for the successful implementation of future HIV therapies.