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

25.4K
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...
25.4K
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

14.0K
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.
14.0K

You might also read

Related Articles

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

Sort by
Same author

Cardiological aspects of Fabry disease: from diagnosis to therapeutic efficacy assessment.

Orphanet journal of rare diseases·2026
Same author

Cardiomyopathy in glycogen storage diseases: diagnosis, prognosis, and advanced management.

Heart failure reviews·2026
Same author

Natural History of Asymptomatic Phenotypically Mild HCM: Insights From the SHaRe Registry.

Journal of the American College of Cardiology·2026
Same author

Multicenter cohort analysis of cardiac amyloidosis patients treated with heart transplant.

Revista espanola de cardiologia (English ed.)·2026
Same author

A translational model of MASLD-associated HFpEF defines mitochondrial dysfunction and cardiac plasticity during disease progression and regression.

Metabolism: clinical and experimental·2026
Same author

AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich Ataxia: A Nonrandomized Clinical Trial.

JAMA cardiology·2026

Related Experiment Video

Updated: Jul 9, 2025

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model
06:03

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model

Published on: June 11, 2020

9.1K

Gene therapy vector-related myocarditis.

Elizabeth Silver1, Alessia Argiro2, Kimberly Hong3

  • 1Division of Cardiovascular Medicine, Department of Medicine, University of California San Diego, San Diego, CA, United States; School of Medicine, University of Connecticut Health Center, Farmington, CT, United States.

International Journal of Cardiology
|November 29, 2023
PubMed
Summary

Gene therapy using adeno-associated virus (AAV) shows promise for inherited cardiomyopathies but faces challenges. Host immune responses to AAV vectors and gene products can cause toxic effects like myocarditis, requiring further research.

Keywords:
CardiomyopathiesGene therapyMyocarditis

More Related Videos

A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice
06:42

A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice

Published on: April 16, 2018

16.0K
Gene Transfer for Ischemic Heart Failure in a Preclinical Model
07:35

Gene Transfer for Ischemic Heart Failure in a Preclinical Model

Published on: May 15, 2011

12.9K

Related Experiment Videos

Last Updated: Jul 9, 2025

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model
06:03

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model

Published on: June 11, 2020

9.1K
A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice
06:42

A Simple and Efficient Method for In Vivo Cardiac-specific Gene Manipulation by Intramyocardial Injection in Mice

Published on: April 16, 2018

16.0K
Gene Transfer for Ischemic Heart Failure in a Preclinical Model
07:35

Gene Transfer for Ischemic Heart Failure in a Preclinical Model

Published on: May 15, 2011

12.9K

Area of Science:

  • Molecular Biology
  • Immunology
  • Cardiology

Background:

  • Gene therapy offers a potential cure for genetic disorders by correcting faulty genes.
  • Adeno-associated virus (AAV)-mediated gene replacement is a leading strategy in clinical trials for inherited cardiomyopathies.
  • Host immune responses to AAV vectors and therapeutic gene products present significant safety and efficacy hurdles.

Purpose of the Study:

  • To review the immunological reactions elicited by adeno-associated virus (AAV)-based gene therapy.
  • To explore the potential toxic effects associated with these immune responses, particularly myocarditis.
  • To discuss future directions and strategies for overcoming immunological challenges in AAV gene therapy.

Main Methods:

  • Literature review of studies investigating immune responses to AAV gene therapy.
  • Analysis of clinical trial data focusing on safety and efficacy of AAV-mediated therapies.
  • Examination of immunological mechanisms underlying AAV vector and transgene product recognition.

Main Results:

  • Host immune responses, including T-cell and B-cell activation, are frequently observed following AAV gene therapy administration.
  • These immunological reactions can lead to reduced therapeutic efficacy and adverse events, such as inflammation and myocarditis.
  • The immunogenicity of both the AAV capsid and the introduced transgene product contributes to the overall immune challenge.

Conclusions:

  • Immune responses remain a critical barrier to the widespread success of AAV-based gene therapy.
  • Understanding and mitigating these reactions are essential for improving the safety and effectiveness of gene replacement therapies for inherited cardiomyopathies.
  • Future research should focus on developing immunomodulatory strategies and optimizing AAV vector design to minimize adverse immunological outcomes.