Регуляция экспрессии целевых генов как прорывное направление в лечении сердечно-сосудистых за6олеваний: в фокусе РНК-терапия

Недавние достижения в области получения, очистки и клеточной доставки PНК в организм пациента позволили разработать терапевтические средства на основе PНК для лечения широкого спектра заболеваний, в том числе и сердечно-сосудистых. PНК-терапия представляет собой новое, быстро развивающееся направление медицины, которое использует в качестве терапевтического средства различные молекулы PНК. Эти препараты экономически эффективны, относительно просты в производстве и могут воздействовать на ранее не поддающиеся медикаментозному лечению патологические процессы. B настоящее время все PНК-препараты подразделены на 5 групп и включают: антисмысловые олигонуклеотиды (ACO) — antisense oligonucleotides (ASO); малые интерферирующие PНК (сиPНК) — small interfering RNAs (siRNAs); микроPНК (миPНК) — microRNAs (miRNAs); PНК-аптамеры (RNA aptamers) и мPНК (mRNAs). PНК-терапевтические препараты предназначены для регуляции активности генов и, в зависимости от избранной стратегии, могут заменять, дополнять, исправлять, подавлять или устранять экспрессию целевого гена. B данном мини-обзоре рассматриваются проблемы и преимущества, связанные с использованием препаратов на основе PНК, различные подходы к их доставке в клетки пациента, а также механизмы действия отдельных PНК-препаратов. Кроме того, приведены сведения об эффективности некоторых препаратов на основе PНК, которые в настоящее время проходят клинические испытания или уже получили одобрение регулирующих органов.

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