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| 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 | 812 | 2018 |
| Silencing primary dystonia: lentiviral-mediated RNA interference therapy for DYT1 dystonia P Gonzalez-Alegre, N Bode, BL Davidson, HL Paulson Journal of Neuroscience 25 (45), 10502-10509, 2005 | 130 | 2005 |
| AsCas12a ultra nuclease facilitates the rapid generation of therapeutic cell medicines L Zhang, JA Zuris, R Viswanathan, JN Edelstein, R Turk, B Thommandru, ... Nature communications 12 (1), 3908, 2021 | 120 | 2021 |
| Dorsal raphe serotonin neurons mediate CO2-induced arousal from sleep HR Smith, NK Leibold, DA Rappoport, CM Ginapp, BS Purnell, NM Bode, ... Journal of Neuroscience 38 (8), 1915-1925, 2018 | 87 | 2018 |
| Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells MK Cromer, J Camarena, RM Martin, BJ Lesch, CA Vakulskas, NM Bode, ... Nature medicine 27 (4), 677-687, 2021 | 77 | 2021 |
| Disruption of protein processing in the endoplasmic reticulum of DYT1 knock-in mice implicates novel pathways in dystonia pathogenesis G Beauvais, NM Bode, JL Watson, H Wen, KA Glenn, H Kawano, ... Journal of Neuroscience 36 (40), 10245-10256, 2016 | 48 | 2016 |
| Human genetics and neuropathology suggest a link between miR-218 and amyotrophic lateral sclerosis pathophysiology I Reichenstein, C Eitan, S Diaz-Garcia, G Haim, I Magen, A Siany, ... Science translational medicine 11 (523), eaav5264, 2019 | 46 | 2019 |
| Transcriptional and proteomic profiling in a cellular model of DYT1 dystonia JN Martin, TB Bair, N Bode, WT Dauer, P Gonzalez-Alegre Neuroscience 164 (2), 563-572, 2009 | 33 | 2009 |
| Comparative analysis of CRISPR off-target discovery tools following ex vivo editing of CD34+ hematopoietic stem and progenitor cells MK Cromer, KR Majeti, GR Rettig, K Murugan, GL Kurgan, NM Bode, ... Molecular Therapy 31 (4), 1074-1087, 2023 | 19 | 2023 |
| The ubiquitin ligase F-box/G-domain protein 1 promotes the degradation of the disease-linked protein torsinA through the ubiquitin–proteasome pathway and macroautophagy KL Gordon, KA Glenn, N Bode, HM Wen, HL Paulson, P Gonzalez-Alegre Neuroscience 224, 160-171, 2012 | 11 | 2012 |
| DYT1 knock-in mice are not sensitized against mitochondrial complex-II inhibition N Bode, C Massey, P Gonzalez-Alegre Public Library of Science 7 (8), e42644, 2012 | 10 | 2012 |
| Engineered AsCas12a Variants with Enhanced Activity and Broadened PAM Compatibility MA Collingwood, CA Vakulskas, L Zhang, R Turk, NM Bode, AM Jacobi, ... Journal of Biomolecular Techniques: JBT 30 (Suppl), S49, 2019 | 9 | 2019 |
| Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency M Pavel-Dinu, CL Gardner, Y Nakauchi, T Kawai, OM Delmonte, ... Blood Advances 8 (7), 1820-1833, 2024 | 7 | 2024 |
| Novel mutations that enhance the dna cleavage activity of acidaminococcus sp. cpf1 L Zhang, CA VAKULSKAS, NM Bode, MA Collingwood, KR BELTZ, ... | 6* | 2020 |
| Cas12a Mutant Genes and Polypeptides Encoded by Same CA Vakulskas, NM Bode, MA Collingwood, S Beaudoin US Patent App. 16/735,841, 2020 | 3 | 2020 |
| S. pyogenes Cas9 mutant genes and polypeptides encoded by same CA Vakulskas, NM Bode, MA Collingwood, GR Rettig, MA Behlke US Patent 11,242,542, 2022 | 1 | 2022 |
| S. pyogenes cas9 mutant genes and polypeptides encoded by same CA Vakulskas, NM Bode, MA Collingwood, GR Rettig, MA Behlke US Patent App. 18/418,248, 2024 | | 2024 |
| S. pyogenes Cas9 mutant genes and polypeptides encoded by same CA Vakulskas, NM Bode, MA Collingwood, GR Rettig, MA Behlke US Patent 11,913,014, 2024 | | 2024 |
| Ubiquitin variants with improved affinity for 53bp1 CA Vakulskas, NM Bode, SE Glenn, L Zhang US Patent App. 17/952,252, 2023 | | 2023 |
