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Миша Давыдов

19 мая 2020 19.05.2020

CRISPR-Cas9 впервые испытали, введя напрямую в человека

Им стал пациент с наследственным заболеванием сетчатки.

В марте этого года учёные провели клиническое испытание метода редактирования генов CRISPR-Cas9 на способность удалять мутации, которые вызывают редкое состояние, называемое врожденным амаврозом Лебера 10 (LCA10). В настоящее время нет лечения этого заболевания, которое является основной причиной слепоты в детстве.

В этом исследовании компоненты системы редактирования генов, закодированные в геноме вируса, вводятся непосредственно в глаз, рядом с фоторецепторными клетками. Для сравнения, предыдущие клинические испытания CRISPR-Cas9 использовали технику редактирования геномов клеток, которые были удалены заранее из организма. Материал затем вводился обратно в пациента.

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Марат

19.05.2020

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