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Related Concept Videos

Heart Failure Drugs: Inhibitors of Renin-Angiotensin System01:26

Heart Failure Drugs: Inhibitors of Renin-Angiotensin System

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The activation of the sympathetic nervous system and the renin-angiotensin-aldosterone system (RAAS) contributes to cardiac remodeling, and inhibiting the RAAS is a pharmacological target in heart failure management. As a result, neurohumoral modulation is a crucial treatment principle for managing heart failure. This approach involves using medications like ACE inhibitors (ACEIs), angiotensin receptor blockers (ARBs), β-blockers, mineralocorticoid receptor antagonists (MRAs), and neutral...
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Heart Failure V: Medical Management01:30

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Medical Management of Acute Decompensated Heart Failure (ADHF)The primary goals of therapy for patients hospitalized with acute decompensated heart failure (ADHF) include:Relieving symptomsOptimizing volume statusSupporting oxygenation and ventilationMaintaining cardiac output (CO) and end-organ perfusionIdentifying and addressing the cause of ADHFPreventing complicationsProviding patient education on factors precipitating HF exacerbationPlanning for dischargeOngoing monitoring and assessment...
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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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Heart Failure VI: Adjunct Therapies01:22

Heart Failure VI: Adjunct Therapies

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Additional therapies for treating patients with heart failure (HF) may include procedural interventions, supplemental oxygen, the management of sleep disorders, and nutritional therapy.Procedural InterventionsImplantable Cardioverter-Defibrillator: For patients at risk of life-threatening arrhythmias due to severe left ventricular dysfunction, an Implantable Cardioverter-Defibrillator (ICD) can detect and terminate these arrhythmias, preventing sudden cardiac death and improving survival rates.
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Heart Failure II: Pathophysiology01:29

Heart Failure II: Pathophysiology

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Systolic Heart Failure and Compensatory MechanismsSystolic heart failure (also termed HFrEF, Heart Failure with Reduced Ejection Fraction) is the most prevalent type of heart filure. It results in a decreased volume of blood being pumped from the ventricle. The aortic arch and carotid sinuses have baroreceptors that detect reduced blood pressure, triggering the sympathetic nervous system (SNS) to release epinephrine and norepinephrine. Initially, this response aims to boost heart rate and...
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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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Related Experiment Video

Updated: Mar 12, 2026

Gene Transfer for Ischemic Heart Failure in a Preclinical Model
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Gene Therapy in Heart Failure.

Anthony S Fargnoli1, Michael G Katz2, Charles R Bridges2

  • 1Icahn School of Medicine at Mount Sinai, Cardiovascular Research Center, New York, NY, USA. anthony.fargnoli@mssm.edu.

Handbook of Experimental Pharmacology
|November 1, 2016
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising, potentially lifelong treatment for heart failure by targeting myocyte dysfunction. Overcoming challenges in target validation, gene manipulation, vector choice, and delivery is crucial for clinical success.

Keywords:
Gene therapyGene therapy vectorsHeart failureMolecular targetsRoutes of gene delivery

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Area of Science:

  • Cardiovascular Medicine
  • Molecular Biology
  • Biotechnology

Background:

  • Heart failure (HF) poses a significant global health burden, with current treatments lacking efficacy in improving survival and addressing underlying myocyte dysfunction.
  • Existing interventions for HF are often costly and do not offer a sustainable, long-term solution for patients.
  • Gene therapy presents a potentially curative approach for HF, offering permanent management of myocyte dysfunction through targeted genetic modification.

Purpose of the Study:

  • To review key developments in cardiac gene therapy.
  • To outline essential components for successful clinical translation of gene therapy for heart failure.
  • To discuss the latest advancements in pharmacologic gene targets, vectors, and delivery strategies.

Main Methods:

  • Review of current literature on cardiac gene therapy.
  • Analysis of critical elements for successful gene therapy implementation: target validation, gene manipulation, vector selection, and delivery routes.
  • Synthesis of recent findings on pharmacologic gene targets, vectors, and delivery methods.

Main Results:

  • Gene therapy holds promise for sustainable, single-treatment management of heart failure.
  • Successful cardiac gene therapy requires careful consideration of myocyte targets, gene manipulation strategies, vector choice, and efficient, safe delivery routes.
  • Recent progress has been made in identifying novel gene targets, developing advanced vectors, and optimizing delivery systems for cardiac gene therapy.

Conclusions:

  • Cardiac gene therapy is a viable therapeutic strategy for heart failure, addressing root causes of myocyte dysfunction.
  • Addressing challenges in target validation, gene manipulation, vector selection, and delivery is essential for clinical application.
  • Continued research and development in these areas will facilitate the translation of gene therapy into effective HF treatments.