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

[1] TALEN-based gene correction for epidermolysis bullosa. Osborn MJ,Starker CG,McElroy AN,Webber BR,Riddle MJ,Xia L,et al. Molecular Therapy . 2013

[2] Safe harbours for the integration of new DNA in the human genome. Sadelain Michel,Papapetrou Eirini P,Bushman Frederic D. Nature reviews. Cancer . 2011

[3] A transcription activator-like effector toolbox for genome engineering. Sanjana Neville E,Cong Le,Zhou Yang,Cunniff Margaret M,Feng Guoping,Zhang Feng. Nature protocols . 2012

[4] DNA targeting specificity of RNA-guided Cas9 nucleases. Hsu PD,Scott DA,Weinstein JA,Ran FA,Konermann S,Agarwala V,Li YQ,Fine EJ,Wu XB,Shalem O,Cradick TJ,Marraffini LA,Bao G,Zhang F. Nature Biotechnology . 2013

[5] Epigenetics:Modifying chromatin to shut off enhancers. Rusk N. Nat Methods . 2013

[6] Obligate ligation-gated recombination (ObLiGaRe):custom-designed nuclease-mediated targeted integration through nonhomologous end joining. Maresca M,Lin VG,Guo N,et al. Genome Research . 2013

[7] Generation of an HIV resistant T-cell line by targeted"stacking"of restriction factors. Voit RA,McMahon MA,Sawyer SL,et al. Molecular Therapy . 2013

[8] Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Cho SW,Kim S,Kim Y,et al. Genome Research . 2014

[9] Mammalian Gene Targeting with Designed Zinc Finger Nucleases. Matthew H. Porteus. Molecular Therapy . 2006

[10] Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Pattanayak Vikram,Ramirez Cherie L,Joung J Keith,Liu David R. Nature methods . 2011

[1] TALEN-based gene correction for epidermolysis bullosa. Osborn MJ,Starker CG,McElroy AN,Webber BR,Riddle MJ,Xia L,et al. Molecular Therapy . 2013

[2] Safe harbours for the integration of new DNA in the human genome. Sadelain Michel,Papapetrou Eirini P,Bushman Frederic D. Nature reviews. Cancer . 2011

[3] A transcription activator-like effector toolbox for genome engineering. Sanjana Neville E,Cong Le,Zhou Yang,Cunniff Margaret M,Feng Guoping,Zhang Feng. Nature protocols . 2012

[4] DNA targeting specificity of RNA-guided Cas9 nucleases. Hsu PD,Scott DA,Weinstein JA,Ran FA,Konermann S,Agarwala V,Li YQ,Fine EJ,Wu XB,Shalem O,Cradick TJ,Marraffini LA,Bao G,Zhang F. Nature Biotechnology . 2013

[5] Epigenetics:Modifying chromatin to shut off enhancers. Rusk N. Nat Methods . 2013

[6] Obligate ligation-gated recombination (ObLiGaRe):custom-designed nuclease-mediated targeted integration through nonhomologous end joining. Maresca M,Lin VG,Guo N,et al. Genome Research . 2013

[7] Generation of an HIV resistant T-cell line by targeted"stacking"of restriction factors. Voit RA,McMahon MA,Sawyer SL,et al. Molecular Therapy . 2013

[8] Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases. Cho SW,Kim S,Kim Y,et al. Genome Research . 2014

[9] Mammalian Gene Targeting with Designed Zinc Finger Nucleases. Matthew H. Porteus. Molecular Therapy . 2006

[10] Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection. Pattanayak Vikram,Ramirez Cherie L,Joung J Keith,Liu David R. Nature methods . 2011