Gene therapy of cardiomyopathies: opportunities and current perspectives
Abstract
Non-ischemic
(primary) cardiomyopathies (CMP) are a large group of genetically determined
diseases manifested by functional and anatomical remodeling of the heart muscle
in the absence of obvious external causes. CMP are recognized as the most
common hereditary heart disease with a total prevalence 0.5% worldwide.
The natural course of
all primary cardiomyopathies is steadily progressive leading to heart failure
(HF). Currently available treatment of primary CMP is mainly symptomatic and
helps to control various symptoms but does not lead to a complete cure.
Etiological approach for the treatment of inherited CMP focused on genetic
defect correction, is the priority task of the modern medicine. In this paper,
we discuss the main achievements in the gene therapy for primary myocardial
dysfunction that have already been introduced or are close to entering the real
clinical practice.
Keywords:non-ischemic cardiomyopathies; gene therapy; genome editing; RNA interference; antisense oligonucleotide; adenovirus-associated vectors; exon skipping; small interfering RNA; siRNA
Funding. The study was financially supported by the State
Budget Project of the Ministry of Science and Higher Education of the Russian
Federation for Research Centre for Medical Genetics.
Conflict of interest. The authors declare no conflict of interest.
For citation: Lavrov A.V., Zaklyazminskaya E.V. Gene therapy of
cardiomyopathies: opportunities and current perspectives. Clinical and
Experimental Surgery. Petrovsky Journal. 2023; 11 (1): 32–46. DOI: https://doi.org/10.33029/2308-1198-2023-11-1-32-46
(in Russian)
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