Анализ биораспределения аденоассоциированных вирусов 9 и rh.10 серотипов, кодирующих ген арилсульфатазы А, после предварительной иммунизации свиней вирусом 9 серотипа

Лизосомные болезни накопления представляют собой наследственные заболевания, связанные с нарушением функции лизосом, включая работу ферментов, важных для клеточного метаболизма, сигнальных путей и других биологических процессов. Метахроматическая лейкодистрофия (МЛД), входящая в группу лизосомных болезней накопления, вызвана дефицитом арилсульфатазы А (ARSA), что приводит к накоплению сульфатидов и разрушению миелиновых оболочек структур нервной системы. В статье обсуждается эффективность использования аденоассоциированных вирусов (AAВ) при нейродегенеративных заболеваниях, включая МЛД. Показано биораспределение и безопасность векторов AAВ9-ARSA и AAВrh.10-ARSA после предварительной иммунизации свиней. Обсуждены иммунные аспекты, связанные с генной терапией на основе AAВ, а также возможные ограничения в эффективности и долгосрочности такой терапии.

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