Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success.
EMBO Mol Med. 2016 May 2;8(5):439-41. doi: 10.15252/emmm.201606325. Print 2016 May.
EMBO Mol Med. 2016.
PMID: 27138565
Free PMC article.