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Gene Therapy

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 Therapy

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...

Heart Failure II: Pathophysiology

Heart Failure II: Pathophysiology

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...

Heart Failure V: Medical Management

Heart Failure V: Medical Management

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...

Pathophysiology of Heart Failure

Pathophysiology of Heart Failure

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...

Cardiomyopathy II: Dilated Cardiomyopathy

Cardiomyopathy II: Dilated Cardiomyopathy

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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Related Experiment Video

Updated: May 9, 2026

Gene Transfer for Ischemic Heart Failure in a Preclinical Model

Gene Transfer for Ischemic Heart Failure in a Preclinical Model

Published on: May 15, 2011

Progress in gene therapy for heart failure.

Armen Yerevanian¹, Alexan Yerevanian, Roger J Hajjar

¹Cardiovascular Research Center, Mount Sinai School of Medicine, New York, NY.

Journal of Cardiovascular Pharmacology

|August 8, 2013

Summary

Gene therapy offers a promising nonpharmacological treatment for heart failure. Clinical trials using adeno-associated virus 1-SERCA2a show improved outcomes, highlighting gene therapy

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Gene Transfer for Ischemic Heart Failure in a Preclinical Model

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Area of Science:

Cardiology
Molecular Medicine
Biotechnology

Background:

Congestive heart failure (CHF) pathophysiology involves myocardial dysfunction.
Nonpharmacological treatments are needed for heart failure.
Gene therapy targets molecular mechanisms of CHF.

Purpose of the Study:

To explore gene therapy as a treatment for heart failure.
To highlight advancements in vector technology and delivery methods.
To discuss SERCA2a as a therapeutic target.

Main Methods:

Utilizing recombinant adeno-associated viruses for gene transfer.
Employing vascular, pericardial, and direct myocardial delivery approaches.
Investigating SERCA2a gene therapy in clinical trials.

Main Results:

Adeno-associated virus vectors enable safe and efficient gene transfer.
SERCA2a is a promising therapeutic target for heart failure.
Phase 2 trials demonstrated positive outcomes using AAV1-SERCA2a.

Conclusions:

Gene therapy is a viable future therapeutic strategy for CHF.
Advancements in gene therapy vectors and delivery enhance treatment potential.
SERCA2a gene therapy shows significant promise in improving heart failure outcomes.