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Related Concept Videos

Heart Failure Drugs: Inotropic Agents01:26

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Positive inotropic agents are commonly used as the first line of treatment for heart failure. One such agent is digoxin, derived from the genus Digitalis, which has been known for centuries but effectively utilized since 1785. However, these cardiac glycosides can have potentially toxic effects due to their mechanism of action, which involves inhibiting Na+/K+-ATPase and increasing contractility. Digoxin is absorbed orally and distributed in various tissues, including the CNS. It has a long...
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Arrhythmias are irregular heart rhythms occurring when the heart's electrical impulses become abnormal. These disturbances can lead to various symptoms, depending on their severity and the underlying cause. Some common factors contributing to arrhythmias include hypoxia, ischemia, electrolyte imbalances, excessive catecholamine exposure, drug toxicity, and muscle overstretching. Arrhythmias can be classified into two main types based on the rate and site of origin of abnormal heart rhythms.
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Cardiomyopathy III: Hypertrophic Cardiomyopathy01:29

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Hypertrophic cardiomyopathy, or HCM, is an autosomal dominant genetic disorder characterized by asymmetric left ventricular hypertrophy without ventricular dilation. It is more common in men and is typically diagnosed in young, athletic adults.EtiologyHCM is primarily genetic and is caused by mutations in genes encoding sarcomeric proteins. Researchers have identified over 1400 mutations across at least 11 different genes. Among these, the most frequently occurring mutations are found in the...
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Gene Therapy00:59

Gene Therapy

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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...
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Cardiomyopathy V: Interprofessional Care01:29

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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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Cardiomyopathy II: Dilated Cardiomyopathy01:30

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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,...
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Viral Transgene Expression in Rodent Hearts and the Assessment of Cardiac Arrhythmia Risk
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Gene Therapy for Inherited Cardiac Arrhythmias.

Mara R Turkieltaub Paredes1,2, Mohit Hulsurkar1,2, Xander H T Wehrens3,4,5,6,7,8

  • 1Cardiovascular Research Institute, Baylor College of Medicine, Houston, TX, 77030, USA.

Journal of Cardiovascular Translational Research
|October 30, 2025
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising, potentially curative treatment for inherited cardiac arrhythmias, which are genetic heart rhythm disorders. This approach addresses limitations of conventional treatments and explores gene delivery advancements for conditions like Brugada syndrome.

Keywords:
ArrhythmiasArrhythmogenic cardiomyopathyAtrial fibrillationBrugada syndromeCatecholaminergic polymorphic ventricular tachycardiaGene therapyLong QT syndromeViral vectors

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Area of Science:

  • Cardiology
  • Genetics
  • Molecular Biology

Background:

  • Inherited cardiac arrhythmias are genetic disorders affecting the heart's electrical system.
  • These conditions significantly increase mortality risk due to inherited mutations.
  • Current treatments like drugs and surgery often lack efficacy and cause complications.

Purpose of the Study:

  • To review gene therapy applications for inherited cardiac arrhythmias.
  • To analyze gene delivery methods for treating these heart rhythm disorders.
  • To highlight the therapeutic potential and challenges of gene therapy.

Main Methods:

  • Review of recent studies on gene therapy for specific inherited arrhythmias.
  • Analysis of advancements in viral and non-viral gene delivery systems.
  • Examination of molecular mechanisms and candidate genes.

Main Results:

  • Gene therapy is emerging as a feasible and potentially curative treatment.
  • Elucidation of molecular mechanisms and identification of candidate genes are advancing.
  • Progress in gene delivery methods enhances therapeutic potential.

Conclusions:

  • Gene therapy presents a viable alternative to conventional treatments for inherited cardiac arrhythmias.
  • Further advancements in gene delivery are crucial for therapeutic success.
  • This review covers Brugada syndrome, CPVT, LQTS, AF, and ACM, assessing gene therapy's role.