Current Epigenetic Treatments

Year 2024, , 111 - 118, 01.10.2024

Didem Seven

https://doi.org/10.59518/farabimedj.1542765

Abstract

The epigenetic mechanisms are defined as alterations in gene expression levels through histone modifications, DNA methylations and non-coding RNAs without any change in DNA sequence. Studies conducted at the molecular level have shown that epigenetic regulations are key players in the pathogenesis of many diseases, and effective biomarkers have been identified in the diagnosis and prognosis of some diseases. While the research of the identified biomarkers in clinical trials carry on, some epigenetic treatment tools are started to be used in treatment with FDA approval. Histone deacetylase inhibitors, DNA methyl transferase inhibitors and non-coding RNAs are used for therapeutic purposes in the pathogenesis of many diseases, especially cancer. Although it took time for epigenetic therapies to be approved, their effectiveness has been shown and is now applied in the clinic as a single or combined therapy. For this reason, it is important to elucidate and investigate epigenetic targets and their functions and cross-talk between epigenetic mediator, modulator and regulator proteins. This review will summarise the epigenetic therapies that are used in routine clinical practice, have received FDA approval, and are frequently used in clinical trials.

Keywords

FDA approved epigenetic therapies, DNA methyl transferase inhibitors, siRNA drugs., Histone deacetylase inhibitors

Güncel Epigenetik Tedaviler

Abstract

Epigenetik mekanizmalar, histon modifikasyonları, DNA metilasyonları ve kodlanmayan RNA’lar aracılığı ile DNA dizisinde herhangi bir değişiklik göstermeksizin, gen ifade düzeylerinin değişmesi olarak tanımlanmaktadır. Yapılan araştırmalar, epigenetik değişikliklerin birçok hastalığın patogenezinde etkin olduğunu göstermiş, bazı hastalıkların tanı ve prognozunda etkili biyobelirteçler tespit edilmiştir. Belirlenen biyobelirteçlerin hedeflenerek klinik çalışmalarda araştırılması devam ederken, kimi epigenetik tedavi araçları FDA onayı ile tedavide kullanılmaktadır. Histon deasetilaz inhibitörleri, DNA metil transferaz inhibitörleri ve kodlanmayan RNA’lar başta kanser olmak üzere tekli veya kombine olarak birçok hastalığın patogenezinde tedavi amaçlı kullanılmaktadır. Her ne kadar epigenetik tedavilerin kabul görmesi zaman almış olsa da, etkinliği kanıtlanmıştır ve günümüzde tek veya kombine terapi olarak klinikte uygulanmaktadır. Bu sebeple epigenetik mekanizmaların aydınlatılarak hedeflerinin ve düzenleyicilerinin belirlenmesi ve bu hedeflerin işlevlerinin araştırılması önem arz etmektedir. Bu derlemede rutin klinik uygulamada kullanılan, FDA onayı almış ve klinik araştırmalarda sıklıkla kullanılan epigenetik terapiler özetlenecektir.

Keywords

FDA onaylı epigenetik tedaviler, DNA metil transferaz inhibitörler, siRNA ilaçları., Histon deasetilaz inhibitörleri

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