Методы генной терапии и способы доставки (обзор)

Генная терапия эволюционировала в высокотехнологичную область, охватывающую разнообразные платформы прецизионного редактирования и современные системы доставки, способные лечить сложные генетические нарушения и возрастные патологии. Данный обзор демонстрирует современное состояние генно-терапевтических технологий, включая геномное редактирование на основе CRISPR, системы редактирования оснований, платформы прайм-редактирования и новые редакторы на основе ДНК-полимеразы наряду с соответствующими методами доставки. В обзоре обсуждаются вирусные векторы, включая тканеспецифичные серотипы адено-ассоциированных вирусов; невирусные системы доставки, такие как ионизируемые липидные наночастицы и вирусоподобные частицы; а также инновационные платформы, в том числе доставка на основе экзосом и системa SEND. Рассматриваются терапевтические стратегии, охватывающие редактирование ядерного генома, модификацию митохондриального генома, РНК-редактирование и эпигенетическую модуляцию, что демонстрирует расширение области применения генной терапии за пределы традиционных моногенных нарушений. Критический анализ выявляет серьезные проблемы, которые сохраняются, несмотря на существующие фундаментальные технологические возможности. Главные вызовы связаны с масштабированием производства, долгосрочной оценкой безопасности, преодолением физиологических барьеров при доставке препаратов и повышением эффективности редактирования в постмитотических тканях. Интеграция подходов искусственного интеллекта для прогностического анализа и рационального дизайна векторов представляет перспективное направление для нивелирования текущих ограничений. Данный обзор показывает, что успешная клиническая реализация требует системного решения проблем производства, безопасности и доставки наряду с разработкой стандартизированных протоколов для стратификации пациентов и надежных регуляторных рамок, которые учитывают быстрые технологические инновации при обеспечении безопасности пациентов.

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