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Related Concept Videos

Cardiomyopathy V: Interprofessional Care01:29

Cardiomyopathy V: Interprofessional Care

Managing cardiomyopathy involves addressing underlying or precipitating causes, treating heart failure with medications, and implementing dietary changes and a balanced exercise and rest regimen.Lifestyle ModificationsCardiomyopathy patients should adopt a low-sodium diet to reduce fluid retention and manage heart failure. A personalized exercise and rest plan helps maintain physical fitness without overstraining the heart. Avoiding alcohol and tobacco is essential to prevent further damage to...
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Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
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Dilated cardiomyopathy, or DCM, is a progressive myocardial disorder characterized by ventricular chamber dilation and contractile dysfunction.EtiologyVarious factors can cause DCM, including hypertension and heavy alcohol intake, which contribute to the weakening and enlargement of the heart muscle. Viral infections, such as Coxsackievirus B, adenoviruses, and influenza, can lead to DCM by causing inflammation and damage to heart tissue. Certain chemotherapeutic agents, including daunorubicin,...
Cardiomyopathy I: Introduction and Classification01:25

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Cardiomyopathy, or CMP, is a group of diseases affecting the myocardial structure, impairing its ability to pump blood effectively. This condition can lead to arrhythmias, heart failure, or sudden cardiac death.Cardiomyopathies are classified into primary and secondary categories:Primary Cardiomyopathy refers to conditions involving only the heart muscle that are often idiopathic (of unknown cause) or genetic. They primarily affect the myocardium without the involvement of other systemic...
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

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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...

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Updated: May 12, 2026

Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart
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Published on: September 2, 2021

Gene Therapy for Cardiomyopathy: Tools, Targets, and Trials.

Radhika Agarwal1,2

  • 1Brigham and Women's Hospital, Boston, MA.

Circulation. Heart Failure
|May 11, 2026
PubMed
Summary

Cardiac gene therapy shows promise for treating cardiomyopathies, with early trials demonstrating myocardial expression and biomarker improvements. Overcoming delivery and toxicity challenges is key to realizing curative, precision heart failure treatments.

Keywords:
cardiomyopathiesclinical trialsgene editinggenetic therapygenetic vectorsheart failure

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Area of Science:

  • Cardiovascular Medicine
  • Genomic Medicine
  • Biotechnology

Background:

  • Cardiomyopathy is a major cause of heart failure, stemming from genetic factors.
  • Gene therapy, particularly using adeno-associated viruses (AAV), is emerging as a promising treatment.
  • Early clinical trials show potential for AAV-mediated gene replacement therapy.

Purpose of the Study:

  • To review the current progress and challenges in cardiac gene therapy for cardiomyopathies.
  • To highlight emerging platforms and future directions for precision heart failure therapeutics.
  • To discuss the convergence of genomic diagnosis, molecular correction, and targeted delivery.

Main Methods:

  • Review of current literature on gene therapy for cardiomyopathy.
  • Analysis of early-phase clinical trial data for AAV-mediated gene replacement.
  • Exploration of genome-editing technologies like CRISPR base and prime editing.
  • Discussion of novel delivery strategies and vector engineering approaches.

Main Results:

  • Early clinical trials show promising myocardial transgene expression and improved biomarkers (e.g., NT-proBNP).
  • Genome editing offers potential for durable variant correction without double-strand breaks.
  • Significant translational challenges persist, including efficient cardiomyocyte transduction and vector delivery.
  • Novel strategies are being developed to address delivery constraints and potential toxicities.

Conclusions:

  • Cardiac gene therapy is entering a critical phase with the initiation of first-in-human trials.
  • Addressing delivery, toxicity, and immunogenicity hurdles is essential for therapeutic success.
  • The integration of genomic insights with advanced gene-editing and delivery technologies promises a new era of precision heart failure therapy.
  • Curative, mechanism-directed therapies for inherited and acquired cardiomyopathies are on the horizon.