新一代基因组编辑技术在基因治疗及生物制药领域中的应用

[31] In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. V Sebastiano,ML Maeder,JF Angstman,B Haddad,C Khayter,DT Yeo. Stem Cells . 2011

[32] Targeted genome engineeringin human cells with the Cas9 RNA-guided endonuclease. Cho SW,Kim S,Kim JM,Kim JS. Nature Biotechnology . 2013

[33] RNA-guided human genome engineering via Cas9. Mali,P.,Yang,L.H,Esvelt,K.M,Aach,J,Guell,M,DiCarlo,J.E,Norville,J.E,Church,G.M. Science . 2013

[34] A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Mussolino C,Morbitzer R,Lütge F,et al. Nucleic Acids Research . 2011

[31] In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases. V Sebastiano,ML Maeder,JF Angstman,B Haddad,C Khayter,DT Yeo. Stem Cells . 2011

[32] Targeted genome engineeringin human cells with the Cas9 RNA-guided endonuclease. Cho SW,Kim S,Kim JM,Kim JS. Nature Biotechnology . 2013

[33] RNA-guided human genome engineering via Cas9. Mali,P.,Yang,L.H,Esvelt,K.M,Aach,J,Guell,M,DiCarlo,J.E,Norville,J.E,Church,G.M. Science . 2013

[34] A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity. Mussolino C,Morbitzer R,Lütge F,et al. Nucleic Acids Research . 2011