RNA Temelli Terapötik Yaklaşımlar

Year 2024, Volume: 50 Issue: 2, 317 - 329, 08.10.2024

İsmail Korkmaz , Serdal Arslan

https://doi.org/10.32708/uutfd.1427446

Abstract

RNA temelli terapötikler, RNA moleküllerinin hücresel süreçlerdeki etki mekanizmalarının aydınlatılması ve gelişen teknoloji ile oldukça yüksek potansiyele sahip terapötik stratejileri oluşturmaktadır. Bu stratejiler, birçok hastalığın mekanizması, patofizyolojik süreçleri, teşhisi, tedavisi ve hastalığın önlenmesi konusunda yeni alternatifler sunmaktadır. Ayrıca daha önce ‘‘hedeflenemez’’ olarak bilinen birçok patofizyolojik yollara yeni kapılar açmaktadır. RNA bazlı terapötiklerin sağladığı çeşitli moleküler bazlı ajanlar sayesinde tedavisi yeterli düzeyde olmayan hastalıklara umut verici yeni tedavi yöntemleri geliştirlebilecektir. Günümüzde bilinen 16 adet FDA onaylı RNA terapötik ilaç klinikte kullanılmaktadır. Bunun yanında çok sayıda RNA terapötiği geliştirilme aşamasındadır ve bu durum yakın gelecekte birçok hastalık için yeni tedavi yöntemlerine kapı açacaktır. Bu derleme makalesinde halihazırda kullanılan RNA terapötik stratejilerinin mekanizması, sentezlenmesi, paketlenmesi, hedefe iletimi gibi konular araştırılmıştır ve bunun yanında aday terapötik stratejilere de değinilmiştir.

Keywords

ASO, Oligonükleotid, RNA, siRNA, Terapötik

RNA-Based Therapeutic Approaches

Abstract

RNA-based therapeutics constitute therapeutic strategies with very high potential, thanks to the elucidation of the mechanisms of action of RNA molecules in cellular processes and developing technology. These strategies offer new alternatives regarding the mechanisms, pathophysiological processes, diagnosis, treatment and prevention of many diseases. It also opens new doors to many pathophysiological pathways previously known as "untargetable". Thanks to the various molecular-based agents provided by RNA-based therapeutics, promising new treatment methods can be developed for diseases for which treatment is inadequate. Today, 16 known FDA-approved RNA therapeutic drugs are used in clinics. In addition, many RNA therapeutics are under development, which will open the door to new treatment methods for many diseases in the near future. In this review article, topics such as the mechanism, synthesis, packaging, and target delivery of currently used RNA therapeutic strategies are investigated, and candidate therapeutic strategies are also mentioned.

Keywords

ASO, Oligonucleotide, RNA, siRNA, Therapeutic

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