ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs

• Main Authors: Zhiqing Hu (Author), Miaojin Zhou (Author), Yong Wu (Author), Zhuo Li (Author), Xionghao Liu (Author), Lingqian Wu (Author), Desheng Liang (Author)
• Format: Book
• Published: Elsevier, 2019-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Given that the cDNA of F8 is too large to be packaged into adeno-associated virus (AAV) capsids, gene transfer of some versions of B-domain-deleted F8 (BDD-F8) for hemophilia A (HA) treatment has been attempted with promising results. Here, we describe an efficient gene correction via single-strande...