CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice

Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology. Here, we describe treatment strategies for haemophilia B mice using the clustered regularly interspaced short palindromic repeat...

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Pubblicato in:Sci Rep
Autori principali: Ohmori, Tsukasa, Nagao, Yasumitsu, Mizukami, Hiroaki, Sakata, Asuka, Muramatsu, Shin-ichi, Ozawa, Keiya, Tominaga, Shin-ichi, Hanazono, Yutaka, Nishimura, Satoshi, Nureki, Osamu, Sakata, Yoichi
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2017
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5482879/
https://ncbi.nlm.nih.gov/pubmed/28646206
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-017-04625-5
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