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Российский физиологический журнал им. И.М. Сеченова, 2023, T. 109, № 11, стр. 1567-1583

Экспериментальные модели нарушений ЦНС при развитии болезней лизосомального накопления

А. С. Лебедев 1, 2, 3, М. М. Котова 2, Т. О. Колесникова 2, Д. С. Галстян 1, 3, 4, А. В. Калуев 1, 2, 3, 4, 5, 6, *

1 Научный центр мирового уровня “Центр персонализированной медицины”, Национальный медицинский исследовательский центр им. В.А. Алмазова МЗ РФ
Санкт-Петербург, Россия

2 Направление “Нейробиология”, Научный центр генетики и наук о жизни, Научно-технологический университет “Сириус”
Федеральная территория Сириус, Россия

3 Институт трансляционной биомедицины, Санкт-Петербургский государственный университет
Санкт-Петербург, Россия

4 Российский научный центр радиологии и хирургических технологий им. акад. А.М. Гранова МЗ РФ
Санкт-Петербург, Россия

5 Уральский федеральный университет
Екатеринбург, Россия

6 НИИ нейронаук и медицины
Новосибирск, Россия

* E-mail: avkalueff@gmail.com

Поступила в редакцию 27.03.2023
После доработки 09.08.2023
Принята к публикации 09.08.2023

Аннотация

Болезни лизосомального накопления (БЛН) представляют собой группу орфанных заболеваний, вызванных недостаточностью ферментов лизосом, в результате чего происходит накопление непереваренного материала в клетках и повреждаются ткани. Различаясь по типу накопленного материала (белки, липиды или углеводы), БЛН также чрезвычайно разнообразны по своей клинической картине. При этом наиболее частым проявлением БЛН является повреждение мозга, приводящее к различным неврологическим дисфункциям. К настоящему моменту известно более 70 БЛН, для которых практически не существует эффективной терапии. Настоящий обзор посвящен обсуждению существующих БЛН, их последствий для мозга, а также значению экспериментальных (животных) моделей для выяснения механизмов их патогенеза и поиска новых средств терапии.

Ключевые слова: болезни лизосомального накопления, лизосомы, ферментопатии, нарушения ЦНС, животные модели

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