Gene therapy: A Revolution in Medicine or a Risky Game with our DNA?
Introduction: The aim of this review is to provide an overview of the applications of gene therapies in various diseases and to highlight areas where further research is needed. Materials and methods: A review of chosen literature in the PubMed database was conducted, using the following keyword...
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| Format: | Article |
| Language: | English |
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Nicolaus Copernicus University in Toruń
2025-03-01
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| Series: | Quality in Sport |
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| Online Access: | https://apcz.umk.pl/QS/article/view/58956 |
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| author | Julia Kozakiewicz Aleksandra Okońska Kamil Kościelecki Agnieszka Kalisz Iwona Skorulska Klaudia Mączewska Patrycja Długozima Paulina Grzeszczuk Weronika Grywińska Aleksandra Głowacka |
| author_facet | Julia Kozakiewicz Aleksandra Okońska Kamil Kościelecki Agnieszka Kalisz Iwona Skorulska Klaudia Mączewska Patrycja Długozima Paulina Grzeszczuk Weronika Grywińska Aleksandra Głowacka |
| author_sort | Julia Kozakiewicz |
| collection | DOAJ |
| description |
Introduction: The aim of this review is to provide an overview of the applications of gene therapies in various diseases and to highlight areas where further research is needed.
Materials and methods: A review of chosen literature in the PubMed database was conducted, using the following keywords: ,,gene therapy”, “SMA”, “DMD”, “Parkinson’s Disease”, “Huntington’s disease”
Summary: Gene therapy can be useful in a variety of medical conditions, including Parkinson’s disease, pulmonary hypertension, Wiskott-Aldrich syndrome, spinal muscular atrophy (SMA), Huntington’s disease, Duchenne muscular dystrophy (DMD), and chronic pain management. Different gene therapy approaches, such as viral vector delivery, CRISPR-Cas9 gene editing, exon-skipping therapy, and antisense oligonucleotides (ASOs), have demonstrated potential in treating these diseases.
Conclusions: Gene therapy represents a groundbreaking advancement in medicine, offering hope for diseases that were once considered untreatable. Clinical trials have demonstrated encouraging outcomes in treating neurodegenerative and genetic disorders. Future developments in gene editing tools, improved vector systems, and targeted delivery methods will be crucial in enhancing the efficacy of gene therapy.
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| format | Article |
| id | doaj-art-9cba939b84c344ffaade8074cdfc9923 |
| institution | OA Journals |
| issn | 2450-3118 |
| language | English |
| publishDate | 2025-03-01 |
| publisher | Nicolaus Copernicus University in Toruń |
| record_format | Article |
| series | Quality in Sport |
| spelling | doaj-art-9cba939b84c344ffaade8074cdfc99232025-08-20T01:57:36ZengNicolaus Copernicus University in ToruńQuality in Sport2450-31182025-03-013910.12775/QS.2025.39.58956Gene therapy: A Revolution in Medicine or a Risky Game with our DNA?Julia Kozakiewicz0https://orcid.org/0009-0002-5435-3007Aleksandra Okońska1https://orcid.org/0009-0001-9036-7150Kamil Kościelecki2https://orcid.org/0009-0001-5516-7540Agnieszka Kalisz3https://orcid.org/0009-0001-2996-7691Iwona Skorulska4https://orcid.org/0009-0006-6326-2464Klaudia Mączewska5https://orcid.org/0009-0007-3426-6666Patrycja Długozima6https://orcid.org/0009-0001-4513-2256Paulina Grzeszczuk7https://orcid.org/0009-0003-3522-1567Weronika Grywińska8https://orcid.org/0009-0004-8609-9626Aleksandra Głowacka9https://orcid.org/0009-0000-9389-7731University Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandUniversity Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandVoivodeship Integrated Hospital named after Jedrzej Sniadecki in Bialystok, ul. M. C. Skłodowskiej 26, 15-278 BiałystokUniversity Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandUniversity Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandUniversity Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandUniversity Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandUniversity Clinical Hospital in Bialystok ul. M. C. Skłodowskiej 24a, 15-276 Białystok, PolandIndependent Public Complex of Health Care Facilities in Wyszkow ul. KEN 1, 07-200 Wyszków, PolandIndependent Public Complex of Health Care Facilities in Ostrow Mazowiecka ul. S. Dubois 68, 07-300 Ostrów Mazowiecka Introduction: The aim of this review is to provide an overview of the applications of gene therapies in various diseases and to highlight areas where further research is needed. Materials and methods: A review of chosen literature in the PubMed database was conducted, using the following keywords: ,,gene therapy”, “SMA”, “DMD”, “Parkinson’s Disease”, “Huntington’s disease” Summary: Gene therapy can be useful in a variety of medical conditions, including Parkinson’s disease, pulmonary hypertension, Wiskott-Aldrich syndrome, spinal muscular atrophy (SMA), Huntington’s disease, Duchenne muscular dystrophy (DMD), and chronic pain management. Different gene therapy approaches, such as viral vector delivery, CRISPR-Cas9 gene editing, exon-skipping therapy, and antisense oligonucleotides (ASOs), have demonstrated potential in treating these diseases. Conclusions: Gene therapy represents a groundbreaking advancement in medicine, offering hope for diseases that were once considered untreatable. Clinical trials have demonstrated encouraging outcomes in treating neurodegenerative and genetic disorders. Future developments in gene editing tools, improved vector systems, and targeted delivery methods will be crucial in enhancing the efficacy of gene therapy. https://apcz.umk.pl/QS/article/view/58956gene therapySMADMDParkinson's diseaseHuntington's disease |
| spellingShingle | Julia Kozakiewicz Aleksandra Okońska Kamil Kościelecki Agnieszka Kalisz Iwona Skorulska Klaudia Mączewska Patrycja Długozima Paulina Grzeszczuk Weronika Grywińska Aleksandra Głowacka Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? Quality in Sport gene therapy SMA DMD Parkinson's disease Huntington's disease |
| title | Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? |
| title_full | Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? |
| title_fullStr | Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? |
| title_full_unstemmed | Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? |
| title_short | Gene therapy: A Revolution in Medicine or a Risky Game with our DNA? |
| title_sort | gene therapy a revolution in medicine or a risky game with our dna |
| topic | gene therapy SMA DMD Parkinson's disease Huntington's disease |
| url | https://apcz.umk.pl/QS/article/view/58956 |
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