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

26.4K
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...
26.4K
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

15.5K
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.
15.5K
In-situ Hybridization02:31

In-situ Hybridization

9.9K
In situ hybridization (ISH) is a technique used to detect and localize specific DNA or RNA molecules in cells, tissue, or tissue sections using a labeled probe. The technique was first used in 1969 for the investigation of nucleic acids. It is currently an essential tool in scientific research and clinical settings, especially for diagnostic purposes.
Types of probes and labels
A probe is a complementary strand of DNA or RNA that binds to corresponding nucleotide sequences in a cell. Many...
9.9K
Conservative Site-specific Recombination and Phase Variation02:53

Conservative Site-specific Recombination and Phase Variation

6.3K
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...
6.3K
Transgenic Plants02:50

Transgenic Plants

7.9K
Recombinant DNA technology called transgenesis is often used to add a foreign gene or remove a detrimental gene from an organism. Such genetically modified organisms are called transgenic organisms.
The first-ever transgenic plant was a tobacco plant developed in 1983 that showed resistance against the tobacco mosaic virus. Since then, many transgenic plants have been developed and commercialized for improving the agricultural, ornamental, and horticultural value of a crop plant. Transgenic...
7.9K

You might also read

Related Articles

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

Sort by
Same author

Variant profile of Brazilian patients with Sanfilippo syndrome type B.

Genetics and molecular biology·2026
Same author

Genetic insights into the peoples who shaped the American continent.

Genetics and molecular biology·2026
Same author

Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I.

Pharmaceutics·2026
Same author

Unveiling Mucopolysaccharidosis IIIC in Brazil: Diagnostic Journey and Clinical Features of Brazilian Patients Identified Through the MPS Brazil Network.

Diseases (Basel, Switzerland)·2026
Same author

Molecular Profile of Mucopolysaccharidosis Type I Patients in Brazil.

Journal of inherited metabolic disease·2026
Same author

Comprehensive Characterization of a Cluster of Mucopolysaccharidosis IIIB in Ecuador.

Diagnostics (Basel, Switzerland)·2025

Related Experiment Video

Updated: Nov 6, 2025

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium
10:18

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium

Published on: May 6, 2018

9.5K

In Situ Gene Therapy.

Hallana Souza Santos1, Luiza Rodrigues1, Luisa Natalia Pimentel Vera1

  • 1Laboratório Celulas, tecidos e genes do Hospital de Clinicas de Porto Alegre, R. Ramiro Barcelos 2350, 90035-903, Porto Alegre, RS,Brazil.

Current Gene Therapy
|May 5, 2021
PubMed
Summary
This summary is machine-generated.

In situ gene therapy delivers therapeutic nucleic acids directly to a specific site, offering advantages like fewer side effects and lower costs. This approach shows promise for treating various diseases, with more gene therapy products expected soon.

Keywords:
In situ gene therapygene therapy clinical trials.intra-articular gene therapyintracerebral gene therapyintraocular gene therapyintratumoral gene therapy

More Related Videos

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
08:54

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression

Published on: March 29, 2019

7.2K
Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers
12:30

Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers

Published on: August 12, 2014

10.0K

Related Experiment Videos

Last Updated: Nov 6, 2025

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium
10:18

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium

Published on: May 6, 2018

9.5K
In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
08:54

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression

Published on: March 29, 2019

7.2K
Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers
12:30

Vascular Gene Transfer from Metallic Stent Surfaces Using Adenoviral Vectors Tethered through Hydrolysable Cross-linkers

Published on: August 12, 2014

10.0K

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Medical Science

Background:

  • Gene therapy involves delivering nucleic acids to cells for therapeutic effects.
  • In situ gene therapy administers gene products to a specific anatomical site.

Purpose of the Study:

  • To review applications of in situ gene therapy for diverse diseases.
  • To highlight advantages of in situ gene therapy over systemic administration.
  • To discuss the potential of in situ gene therapy as a viable therapeutic strategy.

Main Methods:

  • Review of existing literature on in situ gene therapy.
  • Analysis of different vectors, administration routes, and doses.
  • Examination of clinical trials and approved in situ gene therapy drugs.

Main Results:

  • In situ gene therapy offers reduced side effects, lower vector doses, and decreased costs.
  • Successful applications demonstrated in intra-articular, intraocular, central nervous system, and tumor compartments.
  • Growing number of clinical protocols and positive trial outcomes indicate market expansion.

Conclusions:

  • In situ gene therapy is emerging as a powerful therapeutic approach.
  • The localized delivery method presents significant advantages for various medical conditions.
  • Continued research and development are expected to yield more approved in situ gene therapy products.