Efficient viral delivery of Cas9 into human safe harbor

Abstract Gene editing using CRISPR/Cas9 is a promising method to cure many human genetic diseases. We have developed an efficient system to deliver Cas9 into the adeno-associated virus integration site 1 (AAVS1) locus, known as a safe harbor, using lentivirus and AAV viral vectors, as a step toward...

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Autores principales: Hideki Hayashi, Yoshinao Kubo, Mai Izumida, Toshifumi Matsuyama
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
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Acceso en línea:https://doaj.org/article/fce61c5bca0b424ab95f614d32a7cd92
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