Key result
Novel therapeutic strategies for mitochondrial cardiomyopathy, including gene therapies and stem cells, show promise but remain far from achieving safe and effective clinical therapeutic effects.
Why the study?
The heterogeneity of gene mutation-provoked mitochondrial dysfunction and diverse clinical manifestations hamper the development of effective therapeutics for genetic mitochondrial cardiomyopathy.
This review summarizes the pathogenesis of mitochondrial cardiomyopathies and highlights emerging therapeutic strategies, noting that many remain far from clinical application.
May guide targeted therapies for genetic mitochondrial cardiomyopathy; leaves open clinical translation pending validation.
Genetic mitochondrial cardiomyopathy (MCM) is a myocardial condition characterized by abnormal cardiac structure and function secondary to genetic defects. Severity can vary from asymptomatic to severe with manifestations that include heart failure, arrhythmias, and sudden cardiac death. The heterogeneity of gene mutation-provoked mitochondrial dysfunction and a variety of manifestations hamper the development of effective therapeutics. In this review, we present the common pathogenesis of mitochondrial cardiomyopathies, and the most recent promising novel therapeutic strategies for cardiac repair including use of cells or stem cells, adeno-associated virus (AAV)-based gene therapies, mitochondrial-based therapy, pharmaceutical compounds, dietary supplementation, and exercise. We discuss their respective advantages and limitations that may aid their broader clinical application. Some novel treatments remain a long way from achieving safe and effective clinical therapeutic effects.
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Yang et al. (2023) conducted a review in Genetic mitochondrial cardiomyopathy (MCM). Novel therapeutic strategies (cells, stem cells, AAV-based gene therapies, etc.) was evaluated. Novel therapeutic strategies for mitochondrial cardiomyopathy, including gene therapies and stem cells, show promise but remain far from achieving safe and effective clinical therapeutic effects.
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