Highly efficient endogenous human gene correction using designed zinc-finger nucleases FD Urnov, JC Miller, YL Lee, CM Beausejour, JM Rock, S Augustus, ... Nature 435 (7042), 646-651, 2005 | 2192 | 2005 |
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells A Hendel, RO Bak, JT Clark, AB Kennedy, DE Ryan, S Roy, I Steinfeld, ... Nature biotechnology 33 (9), 985-989, 2015 | 1184 | 2015 |
Chimeric nucleases stimulate gene targeting in human cells MH Porteus, D Baltimore Science 300 (5620), 763-763, 2003 | 1125 | 2003 |
Activation of proto-oncogenes by disruption of chromosome neighborhoods D Hnisz, AS Weintraub, DS Day, AL Valton, RO Bak, CH Li, J Goldmann, ... Science 351 (6280), 1454-1458, 2016 | 1050 | 2016 |
Rapid “open-source” engineering of customized zinc-finger nucleases for highly efficient gene modification ML Maeder, S Thibodeau-Beganny, A Osiak, DA Wright, RM Anthony, ... Molecular cell 31 (2), 294-301, 2008 | 968 | 2008 |
Identification of preexisting adaptive immunity to Cas9 proteins in humans CT Charlesworth, PS Deshpande, DP Dever, J Camarena, VT Lemgart, ... Nature medicine 25 (2), 249-254, 2019 | 949 | 2019 |
Gene targeting using zinc finger nucleases MH Porteus, D Carroll Nature biotechnology 23 (8), 967-973, 2005 | 932 | 2005 |
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells DP Dever, RO Bak, A Reinisch, J Camarena, G Washington, CE Nicolas, ... Nature 539 (7629), 384-389, 2016 | 916 | 2016 |
Transplantation outcomes for severe combined immunodeficiency, 2000–2009 SY Pai, BR Logan, LM Griffith, RH Buckley, RE Parrott, CC Dvorak, ... New England Journal of Medicine 371 (5), 434-446, 2014 | 748 | 2014 |
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells CA Vakulskas, DP Dever, GR Rettig, R Turk, AM Jacobi, MA Collingwood, ... Nature medicine 24 (8), 1216-1224, 2018 | 740 | 2018 |
Newborn screening for severe combined immunodeficiency in 11 screening programs in the United States A Kwan, RS Abraham, R Currier, A Brower, K Andruszewski, JK Abbott, ... Jama 312 (7), 729-738, 2014 | 726 | 2014 |
Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells S Durai, M Mani, K Kandavelou, J Wu, MH Porteus, S Chandrasegaran Nucleic acids research 33 (18), 5978-5990, 2005 | 695 | 2005 |
An erythroid enhancer of BCL11A subject to genetic variation determines fetal hemoglobin level DE Bauer, SC Kamran, S Lessard, J Xu, Y Fujiwara, C Lin, Z Shao, ... Science 342 (6155), 253-257, 2013 | 674 | 2013 |
Spatially restricted expression of Dlx-1, Dlx-2 (Tes-1), Gbx-2, and Wnt-3 in the embryonic day 12.5 mouse forebrain defines potential transverse and longitudinal segmental … A Bulfone, L Puelles, MH Porteus, MA Frohman, GR Martin, ... Journal of Neuroscience 13 (7), 3155-3172, 1993 | 657 | 1993 |
Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells J Zou, ML Maeder, P Mali, SM Pruett-Miller, S Thibodeau-Beganny, ... Cell stem cell 5 (1), 97-110, 2009 | 625 | 2009 |
Use of chimeric nucleases to stimulate gene targeting D Baltimore, M Porteus US Patent 9,447,434, 2016 | 452 | 2016 |
MOF and histone H4 acetylation at lysine 16 are critical for DNA damage response and double-strand break repair GG Sharma, S So, A Gupta, R Kumar, C Cayrou, N Avvakumov, U Bhadra, ... Molecular and cellular biology 30 (14), 3582-3595, 2010 | 355 | 2010 |
Human SMC5/6 complex promotes sister chromatid homologous recombination by recruiting the SMC1/3 cohesin complex to double‐strand breaks PR Potts, MH Porteus, H Yu The EMBO journal 25 (14), 3377-3388, 2006 | 323 | 2006 |
Mutations in the nuclear bile acid receptor FXR cause progressive familial intrahepatic cholestasis N Gomez-Ospina, CJ Potter, R Xiao, K Manickam, MS Kim, KH Kim, ... Nature communications 7 (1), 10713, 2016 | 296 | 2016 |
CRISPR/Cas9 genome editing in human hematopoietic stem cells RO Bak, DP Dever, MH Porteus Nature protocols 13 (2), 358-376, 2018 | 289 | 2018 |