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Research Output

  • 36 Article
  • 4 Review article
  • 2 Chapter
  • 1 Conference contribution
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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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

Comparing genome editing technologies

Gersbach, C. A., Gaj, T. & Barbas, C. F., Mar 1 2014, In : Genetic Engineering and Biotechnology News. 34, 5

Research output: Contribution to journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

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.e4

Research output: Contribution to journalArticle

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 journalArticle

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 journalArticle

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 journalArticle

Open Access
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 journalArticle