| 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 | 721 | 2018 |
| CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery HX Wang, M Li, CM Lee, S Chakraborty, HW Kim, G Bao, KW Leong Chemical Reviews 117 (15), 9874-9906, 2017 | 493 | 2017 |
| COSMID: a web-based tool for identifying and validating CRISPR/Cas off-target sites TJ Cradick, P Qiu, CM Lee, EJ Fine, G Bao Molecular Therapy-Nucleic Acids 3, 2014 | 383 | 2014 |
| CD7-edited T cells expressing a CD7-specific CAR for the therapy of T-cell malignancies D Gomes-Silva, M Srinivasan, S Sharma, CM Lee, DL Wagner, TH Davis, ... Blood 130 (3), 285-296, 2017 | 372 | 2017 |
| Streptococcus thermophilus CRISPR-Cas9 systems enable specific editing of the human genome M Müller, CM Lee, G Gasiunas, TH Davis, TJ Cradick, V Siksnys, G Bao, ... Molecular Therapy 24 (3), 636-644, 2016 | 294 | 2016 |
| The Neisseria meningitidis CRISPR-Cas9 system enables specific genome editing in mammalian cells CM Lee, TJ Cradick, G Bao Molecular Therapy 24 (3), 645-654, 2016 | 257 | 2016 |
| Gene correction for SCID-X1 in long-term hematopoietic stem cells M Pavel-Dinu, V Wiebking, BT Dejene, W Srifa, S Mantri, CE Nicolas, ... Nature communications 10 (1), 1634, 2019 | 206 | 2019 |
| AAV-CRISPR gene editing is negated by pre-existing immunity to Cas9 A Li, MR Tanner, CM Lee, AE Hurley, M De Giorgi, KE Jarrett, TH Davis, ... Molecular Therapy 28 (6), 1432-1441, 2020 | 169 | 2020 |
| Engineered materials for in vivo delivery of genome-editing machinery S Tong, B Moyo, CM Lee, K Leong, G Bao Nature Reviews Materials 4 (11), 726-737, 2019 | 163 | 2019 |
| Efficient CRISPR/Cas9-mediated genome editing using a chimeric single-guide RNA molecule H Butt, A Eid, Z Ali, MAM Atia, MM Mokhtar, N Hassan, CM Lee, G Bao, ... Frontiers in plant science 8, 283573, 2017 | 156 | 2017 |
| Nuclease Target Site Selection for Maximizing On-target Activity and Minimizing Off-target Effects in Genome Editing CM Lee, TJ Cradick, EJ Fine, G Bao Molecular Therapy 24 (3), 475-487, 2016 | 141 | 2016 |
| Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease SH Park, CM Lee, DP Dever, TH Davis, J Camarena, W Srifa, Y Zhang, ... Nucleic acids research 47 (15), 7955-7972, 2019 | 140 | 2019 |
| Correction of the ΔF508 Mutation in the Cystic Fibrosis Transmembrane Conductance Regulator Gene by Zinc-Finger Nuclease Homology-Directed Repair CM Lee, R Flynn, JA Hollywood, MF Scallan, PT Harrison BioResearch Open Access 1 (3), 99-108, 2012 | 138 | 2012 |
| A self-deleting AAV-CRISPR system for in vivo genome editing A Li, CM Lee, AE Hurley, KE Jarrett, M De Giorgi, W Lu, KS Balderrama, ... Molecular Therapy-Methods & Clinical Development 12, 111-122, 2019 | 125 | 2019 |
| Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements A Lattanzi, V Meneghini, G Pavani, F Amor, S Ramadier, T Felix, ... Molecular Therapy 27 (1), 137-150, 2019 | 122 | 2019 |
| Spatial control of in vivo CRISPR–Cas9 genome editing via nanomagnets H Zhu, L Zhang, S Tong, CM Lee, H Deshmukh, G Bao Nature biomedical engineering 3 (2), 126-136, 2019 | 118 | 2019 |
| High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia S Vaidyanathan, AA Salahudeen, ZM Sellers, DT Bravo, SS Choi, ... Cell Stem Cell 26 (2), 161-171. e4, 2020 | 109 | 2020 |
| Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I N Gomez-Ospina, SG Scharenberg, N Mostrel, RO Bak, S Mantri, ... Nature communications 10 (1), 4045, 2019 | 106 | 2019 |
| Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates O Humbert, S Radtke, C Samuelson, RR Carrillo, AM Perez, SS Reddy, ... Science translational medicine 11 (503), eaaw3768, 2019 | 104 | 2019 |
| A Burden of Rare Variants Associated with Extremes of Gene Expression in Human Peripheral Blood J Zhao, I Akinsanmi, D Arafat, TJ Cradick, CM Lee, S Banskota, ... The American Journal of Human Genetics 98 (2), 299-309, 2016 | 104 | 2016 |
