Gene therapy in cardiomyopathies - Review of the latest reports

Introduction and purpose Gene therapy appears to be a promising treatment for patients with cardiomyopathy and innovative therapeutic option. The aim of this review is to present the latest gene therapy technologies, innovations, research in the treatment of cardiomyopathy and analysis of the eth...

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Main Authors: Magdalena Strzelczyk, Gabriela Pabis, Joanna Wziątek, Paula Bętkowska, Iwona Koziołek, Piotr Kowalik, Patrycja Fatyga, Wiktoria Fatyga, Daria Krzyżanowska, Kinga Dowierciał
Format: Article
Language:English
Published: Nicolaus Copernicus University in Toruń 2025-07-01
Series:Quality in Sport
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Online Access:https://apcz.umk.pl/QS/article/view/61280
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author Magdalena Strzelczyk
Gabriela Pabis
Joanna Wziątek
Paula Bętkowska
Iwona Koziołek
Piotr Kowalik
Patrycja Fatyga
Wiktoria Fatyga
Daria Krzyżanowska
Kinga Dowierciał
author_facet Magdalena Strzelczyk
Gabriela Pabis
Joanna Wziątek
Paula Bętkowska
Iwona Koziołek
Piotr Kowalik
Patrycja Fatyga
Wiktoria Fatyga
Daria Krzyżanowska
Kinga Dowierciał
author_sort Magdalena Strzelczyk
collection DOAJ
description Introduction and purpose Gene therapy appears to be a promising treatment for patients with cardiomyopathy and innovative therapeutic option. The aim of this review is to present the latest gene therapy technologies, innovations, research in the treatment of cardiomyopathy and analysis of the ethical issues associated with the use of modern therapies. Material and methods This review is based on recent articles from the years 2020-2025 found in databases such PubMed, Elsevier. Results Gene editing through CRISPR-Cas technology allows for the modification of genetic material by making changes at precise locations what is highly useful in gene therapy. Gene Expression Regulation (RNAi) involves preventing the production of proteins by silencing a faulty gene. Antisense therapy (ASO) uses antisense oligonucleotides to exclude a particular exon and causes the translation process to stop. Gene replacement involves introducing a healthy copy of a mutated gene into the genome along with an appropriate vector. Conclusions Individual technologies in the treatment of cardiomyopathy show potential for treating patients in the future. It is also important to remember the ethical issues when using solutions based on gene therapy. Clinical trials in the future are necessary to further search for solutions for patients with cardiomyopathy.
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institution DOAJ
issn 2450-3118
language English
publishDate 2025-07-01
publisher Nicolaus Copernicus University in Toruń
record_format Article
series Quality in Sport
spelling doaj-art-8a06e395e1814b96aa61c4f65269455e2025-08-20T03:16:42ZengNicolaus Copernicus University in ToruńQuality in Sport2450-31182025-07-014310.12775/QS.2025.43.61280Gene therapy in cardiomyopathies - Review of the latest reportsMagdalena Strzelczyk0https://orcid.org/0009-0005-6312-3002Gabriela Pabis1https://orcid.org/0009-0002-3208-5160Joanna Wziątek 2https://orcid.org/0009-0002-1656-7477Paula Bętkowska 3https://orcid.org/0009-0002-7050-4039Iwona Koziołek 4https://orcid.org/0009-0002-1077-3640Piotr Kowalik 5https://orcid.org/0009-0009-4813-9742Patrycja Fatyga 6https://orcid.org/0009-0001-1277-1246Wiktoria Fatyga 7https://orcid.org/0009-0000-5931-925XDaria Krzyżanowska 8https://orcid.org/0009-0002-5349-5679Kinga Dowierciał 9https://orcid.org/0009-0000-9132-1026Municipal Medical Centre Jonscher in Łódź, Milionowa 14 Street, 93-113 Łódź, Poland The Provincial Hospital, Grunwaldzka 45, 25-736 Kielce, Poland Municipal Medical Centre Jonscher in Łódź, Milionowa 14 Street, 93-113 Łódź, Poland Municipal Medical Centre Jonscher in Łódź, Milionowa 14 Street, 93-113 Łódź, Poland The Provincial Hospital, Grunwaldzka 45, 25-736 Kielce, Poland Medical University of Lodz, Tadeusz Kościuszko Avenue 4, 90–419 Łódź, Poland The Provincial Hospital, Grunwaldzka 45, 25-736 Kielce, Poland Non-public Health Care Facility Prophylactics Wiesława Piotrowska Limited Liability Company, Jana Jeziorańskiego 137, 25-432 Kielce, Poland Municipal Medical Centre Jonscher in Łódź, Milionowa 14 Street, 93-113 Łódź, Poland Independent Public Regional Specialist Hospital in Chełm Ceramiczna 1 Street, 22-100 Chełm, Poland Introduction and purpose Gene therapy appears to be a promising treatment for patients with cardiomyopathy and innovative therapeutic option. The aim of this review is to present the latest gene therapy technologies, innovations, research in the treatment of cardiomyopathy and analysis of the ethical issues associated with the use of modern therapies. Material and methods This review is based on recent articles from the years 2020-2025 found in databases such PubMed, Elsevier. Results Gene editing through CRISPR-Cas technology allows for the modification of genetic material by making changes at precise locations what is highly useful in gene therapy. Gene Expression Regulation (RNAi) involves preventing the production of proteins by silencing a faulty gene. Antisense therapy (ASO) uses antisense oligonucleotides to exclude a particular exon and causes the translation process to stop. Gene replacement involves introducing a healthy copy of a mutated gene into the genome along with an appropriate vector. Conclusions Individual technologies in the treatment of cardiomyopathy show potential for treating patients in the future. It is also important to remember the ethical issues when using solutions based on gene therapy. Clinical trials in the future are necessary to further search for solutions for patients with cardiomyopathy. https://apcz.umk.pl/QS/article/view/61280cardiomyopathygene editingantisense therapygene expressiongene replacementethical issues
spellingShingle Magdalena Strzelczyk
Gabriela Pabis
Joanna Wziątek
Paula Bętkowska
Iwona Koziołek
Piotr Kowalik
Patrycja Fatyga
Wiktoria Fatyga
Daria Krzyżanowska
Kinga Dowierciał
Gene therapy in cardiomyopathies - Review of the latest reports
Quality in Sport
cardiomyopathy
gene editing
antisense therapy
gene expression
gene replacement
ethical issues
title Gene therapy in cardiomyopathies - Review of the latest reports
title_full Gene therapy in cardiomyopathies - Review of the latest reports
title_fullStr Gene therapy in cardiomyopathies - Review of the latest reports
title_full_unstemmed Gene therapy in cardiomyopathies - Review of the latest reports
title_short Gene therapy in cardiomyopathies - Review of the latest reports
title_sort gene therapy in cardiomyopathies review of the latest reports
topic cardiomyopathy
gene editing
antisense therapy
gene expression
gene replacement
ethical issues
url https://apcz.umk.pl/QS/article/view/61280
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