Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

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...
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...
Reporter Genes02:11

Reporter Genes

Reporter genes are a type of protein-coding gene that are often tagged to a gene of interest. Once inside a target cell, reporter genes usually produce visually identifiable characteristics like fluorescence and luminescence when expressed along with the gene of interest. Thus, reporter genes “report” the presence or absence of genes of interest in an organism, determine the gene expression pattern, or track the physical location of a DNA segment or protein in the cell.
Commonly used reporter...
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

[Ethical challenges and prospective strategies in orthopedic robotic-assisted surgery].

Zhonghua wai ke za zhi [Chinese journal of surgery]·2026
Same author

Lung mTOR activation leads to lung fibrosis or emphysema via senescence of specific lung cells.

Scientific reports·2026
Same author

[Application and pregnancy outcomes analysis of hysteroscopy combined with hysterosalpingo-contrast sonography in intrauterine insemination].

Zhonghua fu chan ke za zhi·2025
Same author

Walking elicits muscle functional changes in the pectoral fin of Polypterus senegalus.

The Journal of experimental biology·2025
Same author

Experimental Observation of the Motion of Ions in a Resonantly Driven Plasma Wakefield Accelerator.

Physical review letters·2025
Same author

[Senescence of the pulmonary endothelial cells: VEGF, a new target in pulmonary pathologies and aging].

Revue des maladies respiratoires·2025

Related Experiment Video

Updated: May 23, 2026

Applying an Inducible Expression System to Study Interference of Bacterial Virulence Factors with Intracellular Signaling
08:51

Applying an Inducible Expression System to Study Interference of Bacterial Virulence Factors with Intracellular Signaling

Published on: June 25, 2015

A calcium-sensitive promoter construct for gene therapy.

E Merlet1, L Lipskaia, A Marchand

  • 1Transatlantic Cardiovascular Research Center, INSERM UMRS 956, UPMC-Paris 6, Paris, France.

Gene Therapy
|March 30, 2012
PubMed
Summary

A novel nuclear factor of activated T cells (NFAT) promoter enhances gene therapy specificity. This targeted approach, activated by calcium, restricts transgene expression to diseased cells, showing promise for vascular proliferative diseases.

More Related Videos

An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness
06:21

An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness

Published on: May 7, 2018

Generating Transgenic Plants with Single-copy Insertions Using BIBAC-GW Binary Vector
12:08

Generating Transgenic Plants with Single-copy Insertions Using BIBAC-GW Binary Vector

Published on: March 28, 2018

Related Experiment Videos

Last Updated: May 23, 2026

Applying an Inducible Expression System to Study Interference of Bacterial Virulence Factors with Intracellular Signaling
08:51

Applying an Inducible Expression System to Study Interference of Bacterial Virulence Factors with Intracellular Signaling

Published on: June 25, 2015

An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness
06:21

An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness

Published on: May 7, 2018

Generating Transgenic Plants with Single-copy Insertions Using BIBAC-GW Binary Vector
12:08

Generating Transgenic Plants with Single-copy Insertions Using BIBAC-GW Binary Vector

Published on: March 28, 2018

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Cardiovascular Research

Background:

  • Targeting diseased cells specifically remains a significant challenge in developing effective pharmacological and biological therapeutics.
  • Gene therapy offers a promising approach for rare diseases and conditions lacking alternative treatments, but off-target effects limit its clinical application.
  • Enhancing gene transfer specificity to target organs is crucial for improving gene therapy safety and efficacy.

Purpose of the Study:

  • To develop a novel promoter for targeted gene expression in diseased cells.
  • To evaluate the efficiency and specificity of a new promoter containing nuclear factor of activated T cells (NFAT) consensus sequences.
  • To assess the therapeutic potential of this promoter in a preclinical model of vascular disease.

Main Methods:

  • Construction and characterization of a novel promoter with six NFAT consensus sequences.
  • In vitro and in vivo assessment of promoter-driven gene expression in vascular smooth muscle cells.
  • Evaluation of transgene expression in response to calcium signaling and endoplasmic reticulum depletion.
  • Preclinical testing of gene therapy using the NFAT promoter to deliver sarco/endoplasmic reticulum (SR/ER) Ca(2+) ATPase 2a in a rat model of restenosis.

Main Results:

  • The novel NFAT promoter demonstrated comparable efficiency to the cytomegalovirus (CMV) promoter in driving gene expression in vascular smooth muscle cells.
  • Transgene expression driven by the NFAT promoter was activated in a calcium-dependent manner following endoplasmic reticulum depletion.
  • Crucially, the NFAT promoter restricted transgene expression exclusively to proliferative and diseased cells, unlike the CMV promoter.
  • Overexpression of sarco/endoplasmic reticulum (SR/ER) Ca(2+) ATPase 2a under the NFAT promoter significantly inhibited restenosis after angioplasty in rats.

Conclusions:

  • The developed NFAT promoter offers a highly specific tool for gene therapy, enabling transgene expression predominantly in diseased cells.
  • This promoter's calcium-dependent activation mechanism provides an additional layer of control, minimizing off-target effects.
  • The NFAT promoter holds significant potential for therapeutic applications in vascular proliferative diseases and other conditions characterized by NFAT pathway upregulation.