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Related Concept Videos

Gene Therapy00:59

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...
Retrovirus Life Cycles01:10

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...
Inhibitors of Virion Maturation and Assembly01:19

Inhibitors of Virion Maturation and Assembly

As part of their replication cycle, certain viruses synthesize long precursor proteins called polyproteins within infected host cells. In human immunodeficiency virus (HIV), two major polyproteins are produced: Gag and Gag-Pol. The Gag polyprotein supplies the structural components of the virus, while Gag-Pol includes essential viral enzymes such as reverse transcriptase, integrase, and protease. After synthesis, these polyproteins move to the host cell membrane, where they assemble into an...
Inhibitors of Viral Protein Synthesis01:30

Inhibitors of Viral Protein Synthesis

Protein synthesis is indispensable for viral replication, as viruses lack the cellular machinery required for this process and must hijack the host's translational apparatus. In response, host cells deploy a critical innate immune defense involving interferons, specialized cytokines that play a central role in inhibiting viral propagation.Upon viral detection, infected cells release interferons that bind to receptors on adjacent uninfected cells, activating the JAK-STAT signaling pathway and...

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Related Experiment Video

Updated: Jun 13, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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 in HIV-infected cells to decrease viral impact by using an alternative delivery method.

Teresa Gonzalo1, Maria Isabel Clemente, Louis Chonco

  • 1Laboratory of Molecular Immunobiology, Plataforma de Laboratorio, Hospital General Universitario Gregorio Marañón, 28007 Madrid, Spain.

Chemmedchem
|April 24, 2010
PubMed
Summary

NN16 dendrimers efficiently deliver genetic material into various cells relevant to HIV infection with minimal toxicity. This non-viral vector shows promise for gene therapy by enhancing HIV inhibition and targeting specific genes in infected cells.

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Last Updated: Jun 13, 2026

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Development of Cell-type specific anti-HIV gp120 aptamers for siRNA delivery
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Development of Cell-type specific anti-HIV gp120 aptamers for siRNA delivery

Published on: June 23, 2011

Area of Science:

  • Biomaterials Science
  • Gene Therapy
  • Virology

Background:

  • Human Immunodeficiency Virus (HIV) infection impacts various cell types, necessitating effective gene therapy vectors.
  • Non-viral vectors are explored for gene delivery due to safety concerns with viral methods.
  • Dendrimers offer unique properties for complexing and delivering genetic material.

Purpose of the Study:

  • To evaluate the transfection ability and biomedical applications of dendrimer NN16 for HIV gene therapy.
  • To assess the toxicity profile of NN16 across diverse cell types relevant to HIV infection.
  • To investigate NN16's efficacy in delivering genetic material for HIV replication inhibition and gene expression modulation.

Main Methods:

  • NN16 dendrimer characterization and toxicity assessment (mitochondrial activity, LDH release, hemolysis, gene expression).
  • Cellular uptake studies using flow cytometry and confocal microscopy.
  • Transfection efficiency and gene knockdown evaluation with antisense oligonucleotides and small interfering RNA (siRNA).

Main Results:

  • NN16 exhibited very low cytotoxicity in various HIV-relevant cells and erythrocytes.
  • High transfection efficiency of genetic material was observed in all tested cell types.
  • NN16 enhanced HIV protein 24 antigen reduction and specifically inhibited cyclooxygenase-2 gene expression in HIV-infected cells.

Conclusions:

  • NN16 dendrimers are effective non-viral vectors for transfecting genetic material into diverse cells relevant to HIV pathology.
  • The combination of high efficacy and low toxicity positions NN16 as a promising candidate for HIV gene therapy.
  • NN16 demonstrates potential for direct or indirect inhibition of HIV replication and targeted gene modulation in infected cells.