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

25.7K
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...
25.7K
What is Genetic Engineering?00:49

What is Genetic Engineering?

74.5K
Overview
74.5K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

106
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.
106
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

14.1K
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.
14.1K
Introduction to Nuclear Reprogramming01:14

Introduction to Nuclear Reprogramming

2.0K
Nuclear reprogramming is the process of switching gene expression of one cell type to that of another cell type, usually from a differentiated cell state to an undifferentiated cell state. Differentiation occurs during processes such as development and morphogenesis, tissue regeneration, and malignancy. Cells can also be artificially induced to reprogram their gene expression by techniques such as nuclear transfer, induced pluripotency, and cell fusion. Such techniques have many applications in...
2.0K
Transgenic Organisms00:53

Transgenic Organisms

31.5K
Overview
31.5K

You might also read

Related Articles

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

Sort by
Same author

Large-scale functional annotation establishes a reference framework for human <i>LRRK2</i> variants.

medRxiv : the preprint server for health sciences·2026
Same author

Single-cell RNA profiling of oligodendroglial lineage cells derived from iPSCs carrying Parkinson's disease-relevant LRRK2-G2019S mutation.

iScience·2026
Same author

Fossil fuel emissions dominate Northern Hemisphere CO<sub>2</sub> seasonal cycle trends under mitigation scenarios.

Nature communications·2026
Same author

National pathways of land-use CO₂ emissions in the 21<sup>st</sup> century.

Nature communications·2026
Same author

Impact of genetic variants in the longevity gene PPARGC1A and cerebrospinal fluid PPARγ levels on clinical trajectories in Parkinson's disease: Potential biomarkers for neurodegeneration and ageing.

Parkinsonism & related disorders·2026
Same author

Genetic variation in antidiabetic drug targets: associations with Parkinson's disease risk and age at onset.

NPJ Parkinson's disease·2026
Same journal

[Rare hereditary and acquired diseases with parkinson's syndrome].

Fortschritte der Neurologie-Psychiatrie·2026
Same journal

[Digital transformation in multiple sclerosis: Advances in diagnostics, monitoring and patient-centred care].

Fortschritte der Neurologie-Psychiatrie·2026
Same journal

[Dementia with Lewy bodies].

Fortschritte der Neurologie-Psychiatrie·2026
Same journal

[Impact of the PEPP reimbursement system and other developments on inpatient care of general psychiatry patients 2005 to 2022: a retrospective analysis].

Fortschritte der Neurologie-Psychiatrie·2026
Same journal

[The pathology of the brain eating amoeba Naegleria fowleri].

Fortschritte der Neurologie-Psychiatrie·2026
Same journal

[Chronic vestibular syndromes: Correct diagnosis and treatment].

Fortschritte der Neurologie-Psychiatrie·2026
See all related articles

Related Experiment Video

Updated: Aug 9, 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

9.4K

[Introduction to Gene Therapy].

Thomas Gasser1

  • 1Zentrum für Neurologie, Hertie-Institut für klinische Hirnforschung, Abteilung Neurodegenerative Erkrankungen, Tübingen.

Fortschritte Der Neurologie-Psychiatrie
|February 22, 2023
PubMed
Summary
This summary is machine-generated.

Gene therapies, including DNA/RNA modification, show success. Both ex vivo and in vivo methods, like CRISPR-Cas9, are advancing genetic treatments.

More Related Videos

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts
11:11

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

12.9K
Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
09:20

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants

Published on: October 18, 2022

4.7K

Related Experiment Videos

Last Updated: Aug 9, 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

9.4K
Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts
11:11

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

12.9K
Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
09:20

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants

Published on: October 18, 2022

4.7K

Area of Science:

  • Genetics and Molecular Biology
  • Biotechnology
  • Medical Science

Context:

  • Gene therapy research has overcome numerous setbacks.
  • Successful clinical applications of gene modification techniques are emerging.
  • Both ex vivo and in vivo approaches are being utilized.

Purpose:

  • To highlight the recent successes and broad scope of gene therapies.
  • To discuss the direct modification of genetic material on DNA or RNA levels.
  • To categorize various gene manipulation strategies under the umbrella of gene therapy.

Summary:

  • Gene therapies involve direct modification of DNA or RNA.
  • Ex vivo (in vitro) and in vivo (in vivo) strategies are proving successful.
  • CRISPR-Cas9 for DNA alteration and RNA interference are key examples.

Impact:

  • Gene therapy offers new avenues for treating genetic disorders.
  • Advancements in gene editing technologies are accelerating therapeutic development.
  • The broad definition of gene therapy encompasses diverse genetic manipulation techniques.