KayMA, MannoCS, RagniMV, et al.Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet, 2000; 24:257–261.
2.
MannoCS, PierceGF, ArrudaVR, et al.Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med, 2006; 12, 342–347.
3.
LiangP, XuY, ZhangX, et al.CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes. Protein Cell, 2015. [Epub ahead of print]; DOI: 10.1007/s13238-015-0153-5.
4.
FrankelMS and ChapmanAR. Human inheritable genetic modifications. Assessing scientific, ethical, religious, and policy issues. (American Association for the Advancement of Science, Washington, DC). 2000.
5.
NIH-RAC. NIH Guidelines for research involving recombinant or synthetic nucleic acid molecules (NIH Guidelines), 2013. http://osp.od.nih.gov/office-biotechnology-activities/biosafety/nih-guidelines (accessed April25, 2015).