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

28.0K
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...
28.0K
Gene Therapy00:59

Gene Therapy

4.7K
4.7K
What is Genetic Engineering?00:49

What is Genetic Engineering?

81.1K
Overview
81.1K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

1.3K
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.
1.3K
CRISPR01:59

CRISPR

58.8K
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
58.8K
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

17.6K
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.
17.6K

You might also read

Related Articles

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

Sort by
Same author

Paediatric-onset autoimmune cytopenia: How can we reduce the long-term mortality?

British journal of haematology·2026
Same author

Ribosomal RNA processing impairments in a B cell immunodeficient patient with WDR75 variants.

Journal of human immunity·2026
Same author

Pharmacological stabilization of hypoxia-inducible factor 1-α dampens the interferon response and promotes glycolysis in Aicardi-Goutières syndrome.

Nature communications·2026
Same author

Author Correction: Inactivation of cytidine triphosphate synthase 1 prevents fatal auto-immunity in mice.

Nature communications·2025
Same author

Inborn errors of immunity: Manifestation, treatment, and outcome-an ESID registry 1994-2024 report on 30,628 patients.

Journal of human immunity·2025
Same author

Lymphedema in patients with X-linked severe combined immunodeficiency.

The journal of allergy and clinical immunology. Global·2025

Related Experiment Video

Updated: Mar 20, 2026

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

9.8K

Gene therapy: Myth or reality?

Alain Fischer1

  • 1Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, 75015 Paris, France; Immunology and Pediatric Hematology Department, Assistance publique-Hôpitaux de Paris, 75015 Paris, France; Inserm UMR 1163, 75015 Paris, France; Collège de France, 75005 Paris, France.

Comptes Rendus Biologies
|June 5, 2016
PubMed
Summary
This summary is machine-generated.

Gene therapy has shown clinical benefits for specific genetic disorders and cancers over 17 years. Future advances in gene editing and vector development promise broader applications.

Keywords:
Adeno-associated virusesB-cell leukemiaChimeric antigen receptors (CAR)Gene editingGene therapyImmunodéficience combinée sévèreLeucémie des cellules BRetrovirusesRécepteur d’antigène chimériqueRétrovirusSevere combined immunodeficiencyThérapie géniqueVirus adéno-déficientÉdition de gène

More Related Videos

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
09:54

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

8.6K
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

8.2K

Related Experiment Videos

Last Updated: Mar 20, 2026

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

9.8K
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
09:54

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

8.6K
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
11:16

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

8.2K

Area of Science:

  • Medical Science
  • Genetics
  • Biotechnology

Background:

  • Gene therapy has progressed significantly over the past 17 years.
  • Clinical benefits are evident in specific inherited disorders and certain cancers.
  • Favorable settings for gene therapy are identified through pathophysiological studies.

Purpose of the Study:

  • To summarize the current state and achievements of gene therapy.
  • To highlight successful applications of gene therapy in various medical conditions.
  • To project future advancements in gene therapy.

Main Methods:

  • Review of clinical outcomes in gene therapy over 17 years.
  • Identification of medical conditions with successful gene therapy interventions.
  • Analysis of advancements in vector development and gene editing technologies.

Main Results:

  • Successful gene therapy applications include immune system disorders, leukodystrophies, hemoglobinopathies, hemophilia B, and retinal dystrophies.
  • Significant progress has been made in treating B-cell leukemias and lymphomas.
  • Ongoing research in vector development and gene editing shows promise for future breakthroughs.

Conclusions:

  • Gene therapy is a viable, albeit developing, treatment modality.
  • Current successes are concentrated in specific genetic and hematologic conditions.
  • Gene editing and improved vectors are expected to expand gene therapy's reach.