Роль фермента PLA2G15 в патогенезе болезни Ниманна-Пика типа C1: новые терапевтические перспективы
Болезнь Ниманна-Пика типа C1 (NPC1) — лизосомное заболевание с накоплением холестерина и нейродегенерацией. Фермент PLA2G15 гидролизует липид BMP, а его ингибирование снижает патологии и продлевает жизнь в мышиной модели NPC1 [Nature, 2025]. Обзор (PubMed, Scopus) анализирует роль PLA2G15, терапевтический потенциал и ограничения. Генетическое подавление PLA2G15 спасает нейроны и снижает липидное накопление, но клинических данных нет. Статья подчеркивает перспективы таргетной терапии и необходимость аналогов в России.
Объем: 9 страниц, 30 источников, таблица, 2 графика. Оформлена по требованиям "Медицинского вестника"
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