Research Output per year
Research Output
2020
Treatment of a Mouse Model of ALS by In Vivo Base Editing
Lim, C. K. W., Gapinske, M., Brooks, A. K., Woods, W. S., Powell, J. E., Zeballos C., M. A., Winter, J., Perez-Pinera, P. & Gaj, T., Jan 1 2020, (Accepted/In press) In : Molecular Therapy.Research output: Contribution to journal › Article
2019
CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington's Disease Mouse Model
Ekman, F. K., Ojala, D. S., Adil, M. M., Lopez, P. A., Schaffer, D. V. & Gaj, T., Sep 6 2019, In : Molecular Therapy - Nucleic Acids. 17, p. 829-839 11 p.Research output: Contribution to journal › Article
Open Access
2018
A Hypothalamic Switch for REM and Non-REM Sleep
Chen, K. S., Xu, M., Zhang, Z., Chang, W. C., Gaj, T., Schaffer, D. V. & Dan, Y., Mar 7 2018, In : Neuron. 97, 5, p. 1168-1176.e4Research output: Contribution to journal › Article
hPSC-Derived Striatal Cells Generated Using a Scalable 3D Hydrogel Promote Recovery in a Huntington Disease Mouse Model
Adil, M. M., Gaj, T., Rao, A. T., Kulkarni, R. U., Fuentes, C. M., Ramadoss, G. N., Ekman, F. K., Miller, E. W. & Schaffer, D. V., May 8 2018, In : Stem Cell Reports. 10, 5, p. 1481-1491 11 p.Research output: Contribution to journal › Article
The continuously evolving CRISPR barcoding toolbox Jin-Soo Kim
Gaj, T. & Perez-Pinera, P., Sep 25 2018, In : Genome biology. 19, 1, 143.Research output: Contribution to journal › Article
2017
Defined and Scalable Differentiation of Human Oligodendrocyte Precursors from Pluripotent Stem Cells in a 3D Culture System
Rodrigues, G. M. C., Gaj, T., Adil, M. M., Wahba, J., Rao, A. T., Lorbeer, F. K., Kulkarni, R. U., Diogo, M. M., Cabral, J. M. S., Miller, E. W., Hockemeyer, D. & Schaffer, D. V., Jun 6 2017, In : Stem Cell Reports. 8, 6, p. 1770-1783 14 p.Research output: Contribution to journal › Article
In vivo genome editing improves motor function and extends survival in a mouse model of ALS
Gaj, T., Ojala, D. S., Ekman, F. K., Byrne, L. C., Limsirichai, P. & Schaffer, D. V., Dec 2017, In : Science Advances. 3, 12, eaar3952.Research output: Contribution to journal › Article
Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery
Gaj, T., Staahl, B. T., Rodrigues, G. M. C., Limsirichai, P., Ekman, F. K., Doudna, J. A. & Schaffer, D. V., Jun 1 2017, In : Nucleic acids research. 45, 11, e98.Research output: Contribution to journal › Article
2016
Adeno-associated virus-mediated delivery of CRISPR-cas systems for genome engineering in mammalian cells
Gaj, T. & Schaffer, D. V., Nov 2016, In : Cold Spring Harbor protocols. 2016, 11, p. 941-952 12 p.Research output: Contribution to journal › Article
A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons
Tervo, D. G. R., Hwang, B. Y., Viswanathan, S., Gaj, T., Lavzin, M., Ritola, K. D., Lindo, S., Michael, S., Kuleshova, E., Ojala, D., Huang, C. C., Gerfen, C. R., Schiller, J., Dudman, J. T., Hantman, A. W., Looger, L. L., Schaffer, D. V. & Karpova, A. Y., Oct 19 2016, In : Neuron. 92, 2, p. 372-382 11 p.Research output: Contribution to journal › Article
CRISPR-mediated activation of latent HIV-1 expression
Limsirichai, P., Gaj, T. & Schaffer, D. V., Mar 1 2016, In : Molecular Therapy. 24, 3, p. 499-507 9 p.Research output: Contribution to journal › Article
Genome-editing technologies: Principles and applications
Gaj, T., Sirk, S. J., Shui, S. L. & Liu, J., Jan 1 2016, In : Cold Spring Harbor Perspectives in Biology. 8, 12, a023754.Research output: Contribution to journal › Article
Reactivation of latent HIV-1 expression by engineered TALE transcription factors
Perdigão, P., Gaj, T., Santa-Marta, M., Barbas, C. F. & Goncalves, J., Mar 2016, In : PloS one. 11, 3, e0150037.Research output: Contribution to journal › Article
2015
Direct protein delivery to mammalian cells using cell-permeable Cys2-His2Zinc-finger domains
Gaj, T. & Liu, J., Mar 25 2015, In : Journal of Visualized Experiments. 2015, 97, e52814.Research output: Contribution to journal › Article
Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells
Liu, J., Gaj, T., Yang, Y., Wang, N., Shui, S., Kim, S., Kanchiswamy, C. N., Kim, J. S. & Barbas, C. F., Oct 22 2015, In : Nature Protocols. 10, 11, p. 1842-1859 18 p.Research output: Contribution to journal › Article
Improved cell-penetrating zinc-finger nuclease proteins for precision genome engineering
Liu, J., Gaj, T., Wallen, M. C. & Barbas, C. F., Mar 2015, In : Molecular Therapy - Nucleic Acids. 4, 3, p. e232 e232.Research output: Contribution to journal › Article
Redesigning recombinase specificity for safe harbor sites in the human genome
Wallen, M. C., Gaj, T. & Barbas, C. F., Sep 28 2015, In : PloS one. 10, 9, e0139123.Research output: Contribution to journal › Article
2014
Cell-penetrating peptide-mediated delivery of TALEN proteins via bioconjugation for genome engineering
Liu, J., Gaj, T., Patterson, J. T., Sirk, S. J. & Barbas, C. F., Jan 20 2014, In : PloS one. 9, 1, e85755.Research output: Contribution to journal › Article
Comparing genome editing technologies
Gersbach, C. A., Gaj, T. & Barbas, C. F., Mar 1 2014, In : Genetic Engineering and Biotechnology News. 34, 5Research output: Contribution to journal › Article
Enhancing the specificity of recombinase-mediated genome engineering through dimer interface redesign
Gaj, T., Sirk, S. J., Tingle, R. D., Mercer, A. C., Wallen, M. C. & Barbas, C. F., Apr 2 2014, In : Journal of the American Chemical Society. 136, 13, p. 5047-5056 10 p.Research output: Contribution to journal › Article
Expanding the zinc-finger recombinase repertoire: Directed evolution and mutational analysis of serine recombinase specificity determinants
Sirk, S. J., Gaj, T., Jonsson, A., Mercer, A. C. & Barbas, C. F., Apr 2014, In : Nucleic acids research. 42, 7, p. 4755-4766 12 p.Research output: Contribution to journal › Article
Protein delivery using Cys2-His2 zinc-finger domains
Gaj, T., Liu, J., Anderson, K. E., Sirk, S. J. & Barbas, C. F., Aug 15 2014, In : ACS chemical biology. 9, 8, p. 1662-1667 6 p.Research output: Contribution to journal › Article
Regulation of endogenous human gene expression by ligand-inducible TALE transcription factors
Mercer, A. C., Gaj, T., Sirk, S. J., Lamb, B. M. & Barbas, C. F., Oct 17 2014, In : ACS synthetic biology. 3, 10, p. 723-730 8 p.Research output: Contribution to journal › Article
Site-selective labeling of a lysine residue in human serum albumin
Asano, S., Patterson, J. T., Gaj, T. & Barbas, C. F., Sep 4 2014, In : Angewandte Chemie - International Edition. 53, 44, p. 11783-11786 4 p.Research output: Contribution to journal › Article
Synthetic zinc finger proteins: The advent of targeted gene regulation and genome modification technologies
Gersbach, C. A., Gaj, T. & Barbas, C. F., Aug 19 2014, In : Accounts of chemical research. 47, 8, p. 2309-2318 10 p.Research output: Contribution to journal › Article
2013
A comprehensive approach to zinc-finger recombinase customization enables genomic targeting in human cells
Gaj, T., Mercer, A. C., Sirk, S. J., Smith, H. L. & Barbas, C. F., Apr 1 2013, In : Nucleic acids research. 41, 6, p. 3937-3946 10 p.Research output: Contribution to journal › Article
2012
Chimeric TALE recombinases with programmable DNA sequence specificity
Mercer, A. C., Gaj, T., Fuller, R. P. & Barbas, C. F., Nov 1 2012, In : Nucleic acids research. 40, 21, p. 11163-11172 10 p.Research output: Contribution to journal › Article
Targeted gene knockout by direct delivery of zinc-finger nuclease proteins
Gaj, T., Guo, J., Kato, Y., Sirk, S. J. & Barbas, C. F., Aug 1 2012, In : Nature Methods. 9, 8, p. 805-807 3 p.Research output: Contribution to journal › Article
2011
Structure-guided reprogramming of serine recombinase DNA sequence specificity
Gaj, T., Mercer, A. C., Gersbach, C. A., Gordley, R. M. & Barbas, C. F., Jan 11 2011, In : Proceedings of the National Academy of Sciences of the United States of America. 108, 2, p. 510-515 6 p.Research output: Contribution to journal › Article
Structure-guided reprogramming of serine recombinase DNA sequence specificity.
Gaj, T., Mercer, A. C., Gersbach, C. A., Gordley, R. M. & Barbas, C. F., Jan 11 2011, In : Proceedings of the National Academy of Sciences of the United States of America. 108, 2, p. 498-503 6 p.Research output: Contribution to journal › Article
Targeted plasmid integration into the human genome by an engineered zinc-finger recombinase
Gersbach, C. A., Gaj, T., Gordley, R. M., Mercer, A. C. & Barbas, C. F., Sep 1 2011, In : Nucleic acids research. 39, 17, p. 7868-7878 11 p.Research output: Contribution to journal › Article
2010
Directed evolution of an enhanced and highly efficient FokI cleavage domain for zinc finger nucleases
Guo, J., Gaj, T. & Barbas, C. F., Jul 2010, In : Journal of Molecular Biology. 400, 1, p. 96-107 12 p.Research output: Contribution to journal › Article
Directed evolution of recombinase specificity by split gene reassembly
Gersbach, C. A., Gaj, T., Gordley, R. M. & Barbas, C. F., Feb 27 2010, In : Nucleic acids research. 38, 12, p. 4198-4206 9 p., gkq125.Research output: Contribution to journal › Article
2007
Tethering small molecules to a phage display library: Discovery of a selective bivalent inhibitor of protein kinase A
Meyer, S. C., Shomin, C. D., Gaj, T. & Ghosh, I., Nov 14 2007, In : Journal of the American Chemical Society. 129, 45, p. 13812-13813 2 p.Research output: Contribution to journal › Article
The AviD-tag, a NeutrAvidin/avidin specific peptide affinity tag for the immobilization and purification of recombinant proteins
Gaj, T., Meyer, S. C. & Ghosh, I., Nov 1 2007, In : Protein Expression and Purification. 56, 1, p. 54-61 8 p.Research output: Contribution to journal › Article
2006
Highly selective cyclic peptide ligands for neutravidin and avidin identified by phage display
Meyer, S. C., Gaj, T. & Ghosh, I., Jul 1 2006, In : Chemical Biology and Drug Design. 68, 1, p. 3-10 8 p.Research output: Contribution to journal › Article