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Related Concept Videos

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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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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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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Pathophysiology of Heart Failure01:17

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Heart failure (HF) is a progressive syndrome involving ventricles that leads to inadequate cardiac output. It can be classified based on location and output or ejection fraction. Ejection fraction (EF) is an essential measurement in the diagnosis and surveillance of HF. Reduced EF corresponds to systolic heart failure (HFrEF). However, HF with preserved ejection fraction (HFpEF) is becoming increasingly prevalent. Also known as diastolic HF, this form of HF is related to aging. The...
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Heart Failure I: Introduction01:27

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Heart failure refers to a clinical syndrome caused by structural or functional cardiac disorders that prevent the heart from pumping an adequate amount of blood to meet the body's metabolic needs. This condition often arises from myocardial infarction or ischemia, leading to decreased cardiac output, reduced tissue perfusion, impaired gas exchange, fluid volume imbalance, and decreased functional ability.Heart failure can result from disruptions in the mechanisms that regulate cardiac output...
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Heart Failure Drugs: Diuretics01:22

Heart Failure Drugs: Diuretics

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Heart failure and kidney perfusion are interconnected in a complex way. Reduced renal perfusion and venous congestion are two significant factors that contribute to renal dysfunction in heart failure. The kidneys, primarily responsible for fluid balance in the body, are adversely affected due to compromised cardiac output and increased venous pressure. In response to reduced renal perfusion, the kidneys activate neurohumoral mechanisms to restore balance. However, these mechanisms can be...
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Gene Transfer for Ischemic Heart Failure in a Preclinical Model
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Gene Therapy for Heart Failure: New Perspectives.

Khatia Gabisonia1, Fabio A Recchia2,3

  • 1Institute of Life Sciences, Fondazione Toscana Gabriele Monasterio, Scuola Superiore Sant'Anna, Piazza Martiri della Liberta` 33, 56127, Pisa, Italy.

Current Heart Failure Reports
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Summary

Gene therapy offers new hope for heart failure (HF) by targeting molecular mechanisms. Despite early setbacks, ongoing research and novel approaches like microRNA delivery show promising regenerative potential for cardiac repair.

Keywords:
AAVDuchenne cardiomyopathyGene therapyHeart failureHippo pathwaymiRNA

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Area of Science:

  • Cardiovascular Medicine
  • Molecular Biology
  • Biotechnology

Background:

  • Heart failure (HF) pathogenesis is complex, with a significant gap between experimental findings and effective therapeutic options.
  • Current pharmacological treatments for HF are limited in efficacy and often associated with side effects.
  • The need for novel, non-pharmacological strategies has driven research into gene therapy for HF.

Purpose of the Study:

  • To review gene therapy strategies developed to target key pathogenic mechanisms and factors in heart failure.
  • To highlight the necessity of continued research in gene therapy for severe cardiac injury and HF.
  • To discuss the evolution of gene therapy approaches for heart failure treatment.

Main Methods:

  • Review of preclinical and clinical research on gene therapy for heart failure.
  • Analysis of viral vector development for efficient gene delivery.
  • Examination of novel therapeutic targets, including gene repair and microRNA delivery.

Main Results:

  • Early clinical trials, such as those involving sarcoendoplasmic reticulum calcium ATPase gene delivery, have shown mixed results.
  • Advancements in viral vector technology are improving the efficiency and safety of gene delivery.
  • Emerging strategies like in vivo gene repair for heritable HF and microRNA-based cardiac regeneration show significant promise.

Conclusions:

  • Gene therapy for heart failure, while facing challenges, continues to evolve with exciting new perspectives.
  • Novel approaches offer potential for cardiac regeneration and treatment of genetic forms of HF.
  • Continued research is crucial to translate these promising findings into effective clinical therapies for heart failure.