TERAPIA GENOWA CRISPR – POTENCJALNE WYKORZYSTANIE, WYZWANIA I ZAGROŻENIA
Słowa kluczowe:
CRISPR, terapia genowa, medycyna regeneracyjna, bioetykaStreszczenie
Abstrakt:
Postęp w technologii edycji genomu otworzył zupełnie nowe perspektywy w medycynie, w szczególności dzięki systemowi CRISPR-Cas9 (ang. clustered regularly interspaced short palindromic repeat – CRISPR). System ten, wykorzystujący precyzyjne ukierunkowanie endonukleazy Cas9 przez przewodnika RNA, umożliwia modyfikację sekwencji DNA, co stanowi podstawę terapii genowych. W niniejszej pracy omawiamy zasady działania CRISPR, a także metody dostarczania narzędzi edycyjnych do komórek, realizowane m.in. za pomocą wektorów wirusowych (AAV) oraz nanocząsteczek lipidowych (LNP). W niniejszym opracowaniu zestawiliśmy potencjalne zastosowania technologii CRISPR w leczeniu chorób genetycznych, zakaźnych oraz nowotworowych, podkreślając jej potencjał w medycynie personalizowanej. Ponadto omówiliśmy wyzwania i problemy związane z techniczną, jak również etyczno-moralną problematyką wykorzystania terapii CRISPR. Pomimo istniejących ograniczeń, CRISPR-Cas9 prezentuje się jako narzędzie o ogromnym potencjale terapeutycznym, które może zrewolucjonizować podejście do leczenia wielu schorzeń. Poniższa praca stanowi kompleksowy przegląd aktualnego stanu wiedzy, wskazując kierunki dalszych badań nad zwiększeniem efektywności i bezpieczeństwa omawianej technologii.
Słowa kluczowe: CRISPR, terapia genowa, medycyna regeneracyjna, bioetyka
Bibliografia
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“Jahrestagung der Deutschen, Österreichischen und Schweizerischen Gesellschaften für Hämatologie und Medizinische Onkologie, 11. bis 14. Oktober 2024, Basel,” Oncol Res Treat, vol. 47, no. Suppl. 2, pp. 11–354, Oct. 2024, doi: 10.1159/000540557.
Opublikowane
19 czerwca 2025
Prawa autorskie (c) 2025 Martyna Szlenk, Wojciech Wysocki, Adrianna Sobolewska, Alicja Suchocka, Patryk Walocha
Licencja
Utwór dostępny jest na licencji Creative Commons Uznanie autorstwa – Użycie niekomercyjne – Bez utworów zależnych 4.0 Międzynarodowe.
