Najnowsze Trendy w Zastosowaniu Terapii Genowej.

Authors

Maciej Baron, ; Bartosz Bula, ; Małgorzata Wachowicz, ; Andrzej Skrzypiec, ; Kamil Liberka,

Keywords:

Terapia genowa, genetyka, onkologia, kardiologia

Synopsis

Abstract

                One of the most significant examples of personalized medicine is the concept of gene therapy. It is determined as the introduction of genes into a cell, or modification of their expression to obtain a therapeutic effect. The introduction of genes is possible by using particles, known as vectors. Two main groups of vectors may be distinguished: viral and non-viral. Currently, viral vectors are used more frequently. However, the relatively high immune system-inducing potential of the viral vectors is the most notable issue in their application. On the other hand, because the etiology of many diseases lies in genetic defects, gene therapy may be used in numerous branches of medicine. Moreover, the results of recent animal studies are vastly promising. Thus, it is believed that gene therapy may become a relevant solution for various diseases in the future.

               

Abstrakt

                Jednym z najbardziej wyrazistych przykładów prób personalizacji medycyny jest terapia genowa. Definiuje się ją jako wprowadzenie genów do komórki lub modyfikację ich ekspresji w celu uzyskania efektu terapeutycznego. Do wprowadzenia genów do komórki stosuje się wektory, które można podzielić na dwie grupy: wirusowe i  nie-wirusowe. Obecnie najczęściej stosuje się wektory wirusowe, których największą wadą jest relatywnie wysoki potencjał do stymulacji odpowiedzi układu immunologicznego pacjenta. Z drugiej strony, ze względu na fakt, że wiele chorób ma swoje podłoże w defektach genetycznych, terapia genowa może zostać wykorzystana w rozmaitych dziedzinach medycyny. Ponadto, niedawne badania na zwierzętach dały bardzo optymistyczne wyniki, dlatego też naukowcy pokładają w terapii genowej wielkie nadzieje.

References

Goetz LH, Schork NJ. Personalized medicine: motivation, challenges, and progress. FertilSteril. 2018;109(6):952-963. doi:10.1016/j.fertnstert.2018.05.006

National Research Council (US) Committee on A Framework for Developing a New Taxonomy of Disease. Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease. Washington (DC): NationalAcademies Press (US); 2011.

Boyer MS, Widmer D, Cohidon C, et al. Representations of personalised medicine in family medicine: a qualitative analysis. BMC PrimCare. 2022;23(1):37. Published 2022 Mar 1. doi:10.1186/s12875-022-01650-w

Lewandowski P, Goławski M, Baron M, Reichman-Warmusz E, Wojnicz R. A Systematic Review of miRNA and cfDNA as Potential Biomarkers for Liquid Biopsy in Myocarditis and Inflammatory Dilated Cardiomyopathy. Biomolecules. 2022;12(10):1476. Published 2022 Oct 13. doi:10.3390/biom12101476

Lundstrom K. Novel Approaches and Strategies for Biologics, Vaccines and CancerTherapies. Elsevier; San Diego, CA, USA: 2015. New era in gene therapy; pp. 15–37.

“Gene & Cell Therapy FAQs | ASGCT - American Society of Gene & Cell Therapy | ASGCT - American Society of Gene & Cell Therapy”. asgct.org. Retrieved 23 July 2021

Williams DA, Orkin SH. Somatic gene therapy. Current status and future prospects. J Clin Invest. 1986;77(4):1053-1056. doi:10.1172/JCI112403

Strachnan T, Read AP (2004). Human Molecular Genetics (3rd ed.). Garland Publishing. p. 616. ISBN 978-0-8153-4184-0.

Nóbrega C, Mendonça L, Matos CA (2020). A handbook of gene and cell therapy. Cham: Springer. ISBN 978-3-030-41333-0. OCLC 1163431307

“Gene Therapy Clinical Trials Worldwide Database”. The Journal of Gene Medicine. Wiley. June 2016. Archived from the original on 31 July 2020.

Shirley JL, de Jong YP, Terhorst C, Herzog RW. Immune Responses to Viral Gene Therapy Vectors. Mol Ther. 2020;28(3):709-722. doi:10.1016/j.ymthe.2020.01.001

Luiz MT, Dutra JAP, Tofani LB, et al. Targeted Liposomes: A Nonviral Gene Delivery System for Cancer Therapy. Pharmaceutics. 2022;14(4):821. Published 2022 Apr 8. doi:10.3390/pharmaceutics14040821

Szczeklik A., Choroby wewnętrzne. Przyczyny, rozpoznanie i leczenie., wyd. MedycynaPraktyczna, 2006.

Maule G, Arosio D, Cereseto A. Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing. Int J Mol Sci. 2020;21(11):3903. Published 2020 May 30. doi:10.3390/ijms21113903

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group. Cell. 1993;72(6):971-983. doi:10.1016/0092-8674(93)90585-e

Tabrizi SJ, Estevez-Fraga C, van Roon-Mom WMC, et al. Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities. Lancet Neurol. 2022;21(7):645-658. doi:10.1016/S1474-4422(22)00121-1

Yamamoto A, Lucas JJ, Hen R. Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease. Cell. 2000;101(1):57-66. doi:10.1016/S0092-8674(00)80623-6

Sudhakar V, Richardson RM. Gene Therapy for Neurodegenerative Diseases. Neurotherapeutics. 2019;16(1):166-175. doi:10.1007/s13311-018-00694-0

Khan S, Barve KH, Kumar MS. Recent Advancements in Pathogenesis, Diagnostics and Treatment of Alzheimer's Disease. Curr Neuropharmacol. 2020;18(11):1106-1125. doi:10.2174/1570159X18666200528142429

Trivedi A, Hoffman J, Arora R. Gene therapy for atrial fibrillation - How close to clinical implementation?. Int J Cardiol. 2019;296:177-183. doi:10.1016/j.ijcard.2019.07.057

Lodish HF, Berk A, Matsudaira P, Kaiser CA, Krieger M, Scott MP, Zipursky SL, Darnell J (2004). Molecular Cell Biology (5th ed.). New York: W.H. Freeman and Company. pp. 230–31. ISBN 0-7167-4366-3.

Bikou O, Thomas D, Trappe K, et al. Connexin 43 gene therapy prevents persistent atrial fibrillation in a porcine model. Cardiovasc Res. 2011;92(2):218-225. doi:10.1093/cvr/cvr209

Silver E, Argiro A, Hong K, Adler E. Gene therapy vector-related myocarditis. Int J Cardiol. 2024;398:131617. doi:10.1016/j.ijcard.2023.131617

Kumar, V., Abbas, A. K., & Aster, J. C. (2017). Robbins Basic Pathology (10th ed.). Elsevier - Health Sciences Division.

Hoang-Le D, Smeenk L, Anraku I, et al. A Kunjin replicon vector encoding granulocyte macrophage colony-stimulating factor for intra-tumoral gene therapy. Gene Ther. 2009;16(2):190-199. doi:10.1038/gt.2008.169

Niu Z, Bai F, Sun T, et al. Recombinant Newcastle Disease virus Expressing IL15 Demonstrates Promising Antitumor Efficiency in Melanoma Model. Technol Cancer Res Treat. 2015;14(5):607-615. doi:10.7785/tcrt.2012.500414

Arabi F, Mansouri V, Ahmadbeigi N. Gene therapy clinical trials, where do we go? An overview. Biomed Pharmacother. 2022;153:113324. doi:10.1016/j.biopha.2022.113324

Ayala Ceja M, Khericha M, Harris CM, Puig-Saus C, Chen YY. CAR-T cell manufacturing: Major process parameters and next-generation strategies. J Exp Med. 2024;221(2):e20230903. doi:10.1084/jem.20230903

Chen L, Chen F, Li J, et al. CAR-T cell therapy for lung cancer: Potential and perspective. Thorac Cancer. 2022;13(7):889-899. doi:10.1111/1759-7714.14375

Servier Medical Art. Medical Illustrations Database. Available at: https://smart.servier.com. Licensed under CC BY 3.0.

Published

June 19, 2025