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...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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.
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview

You might also read

Related Articles

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

Sort by
Same author

Adopting duplex sequencing technology for genetic toxicity testing: A proof-of-concept mutagenesis experiment with N-ethyl-N-nitrosourea (ENU)-exposed rats.

Mutation research. Genetic toxicology and environmental mutagenesis·2023
Same author

Adopting Duplex Sequencing™ Technology for Genetic Toxicity Testing: A Proof-of-Concept Mutagenesis Experiment with N-Ethyl-N-Nitrosourea (ENU)-Exposed Rats.

bioRxiv : the preprint server for biology·2023
Same author

Sequencing of genes of drug response in tumor DNA and implications for precision medicine in cancer patients.

The pharmacogenomics journal·2023
Same author

Genetic variation determines VEGF-A plasma levels in cancer patients.

Scientific reports·2018
Same author

VHL substrate transcription factor ZHX2 as an oncogenic driver in clear cell renal cell carcinoma.

Science (New York, N.Y.)·2018
Same author

Integrative Genomic Analysis of Cholangiocarcinoma Identifies Distinct IDH-Mutant Molecular Profiles.

Cell reports·2017

Related Experiment Video

Updated: Jun 4, 2026

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

Gene therapy: Have the risks associated with viral vectors been solved?

J Todd Auman

    Current Opinion in Molecular Therapeutics
    |February 2, 2011
    PubMed
    Summary

    Gene therapy offers potential cures for genetic disorders by replacing faulty genes. However, recent findings reveal that even advanced viral vectors carry risks, necessitating careful patient selection for clinical trials to balance benefits against potential harm.

    Area of Science:

    • Biomedical science
    • Molecular biology
    • Genetics

    Background:

    • Gene therapy aims to treat monogenic diseases by correcting defective genes.
    • Historically, gene therapy faced challenges due to adverse events linked to viral vectors.
    • Advancements in viral vector technology were expected to enhance safety profiles.

    Discussion:

    • Recent reports highlight potential risks associated with novel viral vectors used in gene therapy.
    • These findings raise concerns about the safety of current gene therapy approaches.
    • Careful consideration of emerging safety data is crucial for the field's progression.

    Key Insights:

    • Newer viral vectors, despite advancements, may still pose risks to patients.
    • The efficacy and safety balance of gene therapy requires ongoing scrutiny.

    Related Experiment Videos

    Last Updated: Jun 4, 2026

    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

  • Published data indicates a need for reassessment of viral vector safety.

    • Outlook:

    • Future gene therapy development must prioritize robust safety evaluations.
    • Clinical trial recruitment necessitates stringent risk-benefit assessments.
    • Continued research is vital to ensure the safe and effective application of gene therapy for genetic diseases.