Gene Therapy for Heart Failure

Heart failure is the leading cause of hospitalization globally, burdening healthcare systems and the economy. Heart failure is a multifactorial cardiac syndrome, where the heart fails to maintain sufficient cardiac output to support body function. As the population ages, heart failure rates will increase. Current treatments, such as medications and surgery, are not suitable for all patients, creating a need for alternative therapies. Gene therapy offers promising new approaches, with therapeutic angiogenesis, regenerative strategies, and calcium ion regulation as key targets. SERCA2a plays a critical role in calcium regulation, and clinical trials have focused on its potential as a therapeutic agent. VEGF-B, which specifically targets the myocardium, also regulates myocardial metabolism and SERCA2a activity. Although clinical trials have been conducted, results have not consistently replicated pre-clinical success. This review summarizes the current state of gene therapy for heart failure, including therapeutic agents, vectors, delivery methods, and preclinical models.

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Publication Details

Journal
Journal of Cardiovascular Translational Research
Published
2026-02-05
DOI
https://doi.org/10.1007/s12265-025-10736-6
Primary Topic
Virus-based gene therapy research
Type
article
Field-Weighted Citation Impact
0.00

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article

Gene Therapy for Heart Failure

Anni Määttä, Hanna Wang, S Yla-Herttuala
Journal of Cardiovascular Translational Research
Virus-based gene therapy research
article

Gene Therapy for Heart Failure

Anni Määttä, Hanna Wang, S Yla-Herttuala
article en

Abstract

Heart failure is the leading cause of hospitalization globally, burdening healthcare systems and the economy. Heart failure is a multifactorial cardiac syndrome, where the heart fails to maintain sufficient cardiac output to support body function. As the population ages, heart failure rates will increase. Current treatments, such as medications and surgery, are not suitable for all patients, creating a need for alternative therapies. Gene therapy offers promising new approaches, with therapeutic angiogenesis, regenerative strategies, and calcium ion regulation as key targets. SERCA2a plays a critical role in calcium regulation, and clinical trials have focused on its potential as a therapeutic agent. VEGF-B, which specifically targets the myocardium, also regulates myocardial metabolism and SERCA2a activity. Although clinical trials have been conducted, results have not consistently replicated pre-clinical success. This review summarizes the current state of gene therapy for heart failure, including therapeutic agents, vectors, delivery methods, and preclinical models.

Journal of Cardiovascular Translational ResearchVol. 19(1)
University of Eastern Finland (FI), Kuopio University Hospital (FI)
Itä-Suomen Yliopisto, Kuopion Yliopistollinen Sairaala
Openalex Percentile: Top 62%
Virus-based gene therapy research
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