ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering T Gaj, CA Gersbach, CF Barbas III Trends in Biotechnology 31 (7), 397-405, 2013 | 5008 | 2013 |
A designer AAV variant permits efficient retrograde access to projection neurons DGR Tervo, BY Hwang, S Viswanathan, T Gaj, M Lavzin, KD Ritola, ... Neuron 92 (2), 372-382, 2016 | 1260 | 2016 |
Genome-editing technologies: principles and applications T Gaj, SJ Sirk, S Shui, J Liu Cold Spring Harbor Perspectives in Biology 8 (12), a023754, 2016 | 453 | 2016 |
Targeted gene knockout by direct delivery of zinc-finger nuclease proteins T Gaj, J Guo, Y Kato, SJ Sirk, CF Barbas III Nature Methods 9 (8), 805-807, 2012 | 426 | 2012 |
Directed evolution of an enhanced and highly efficient FokI cleavage domain for zinc finger nucleases J Guo, T Gaj, CF Barbas III Journal of Molecular Biology 400 (1), 96-107, 2010 | 298 | 2010 |
Cell-penetrating peptide-mediated delivery of TALEN proteins via bioconjugation for genome engineering J Liu, T Gaj, JT Patterson, SJ Sirk, CF Barbas III PLOS ONE 9 (1), e85755, 2014 | 212 | 2014 |
Treatment of a mouse model of ALS by in vivo base editing CKW Lim, M Gapinske, AK Brooks, WS Woods, JE Powell, CMA Zeballos, ... Molecular Therapy 28 (4), 1177-1189, 2020 | 194 | 2020 |
In vivo genome editing improves motor function and extends survival in a mouse model of ALS T Gaj, DS Ojala, FK Ekman, LC Byrne, P Limsirichai, DV Schaffer Science Advances 3 (12), eaar3952, 2017 | 194 | 2017 |
Chimeric TALE recombinases with programmable DNA sequence specificity AC Mercer, T Gaj, RP Fuller, CF Barbas III Nucleic Acids Research 40 (21), 11163-11172, 2012 | 182 | 2012 |
Synthetic zinc finger proteins: the advent of targeted gene regulation and genome modification technologies CA Gersbach, T Gaj, CF Barbas III Accounts of Chemical Research 47 (8), 2309-2318, 2014 | 168 | 2014 |
CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington’s disease mouse model FK Ekman, DS Ojala, MM Adil, PA Lopez, DV Schaffer, T Gaj Molecular Therapy – Nucleic Acids 17, 829-839, 2019 | 167 | 2019 |
Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells J Liu, T Gaj, Y Yang, N Wang, S Shui, S Kim, CN Kanchiswamy, JS Kim, ... Nature Protocols 10 (11), 1842-1859, 2015 | 163 | 2015 |
A hypothalamic switch for REM and non-REM sleep KS Chen, M Xu, Z Zhang, WC Chang, T Gaj, DV Schaffer, Y Dan Neuron 97 (5), 1168-1176, 2018 | 151 | 2018 |
Genome engineering using adeno-associated virus: basic and clinical research applications T Gaj, BE Epstein, DV Schaffer Molecular Therapy 24 (3), 458-464, 2016 | 150 | 2016 |
CRISPR-mediated activation of latent HIV-1 expression P Limsirichai, T Gaj, DV Schaffer Molecular Therapy 24 (3), 499-507, 2016 | 139 | 2016 |
Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase CA Gersbach, T Gaj, RM Gordley, AC Mercer, CF Barbas III Nucleic Acids Research 39 (17), 7868-7878, 2011 | 132 | 2011 |
Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery T Gaj, BT Staahl, G Rodrigues, P Limsirichai, FK Ekman, JA Doudna, ... Nucleic Acids Research 45 (11), e98, 2017 | 96 | 2017 |
Tethering small molecules to a phage display library: discovery of a selective bivalent inhibitor of protein kinase A SC Meyer, CD Shomin, T Gaj, I Ghosh Journal of the American Chemical Society 129 (45), 13812-13813, 2007 | 89 | 2007 |
Expanding the scope of site‐specific recombinases for genetic and metabolic engineering T Gaj, SJ Sirk, CF Barbas III Biotechnology and Bioengineering 111 (1), 1-15, 2014 | 88 | 2014 |
Structure-guided reprogramming of serine recombinase DNA sequence specificity T Gaj, AC Mercer, CA Gersbach, RM Gordley, CF Barbas III Proceedings of the National Academy of Sciences 108 (2), 498-503, 2011 | 88 | 2011 |