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Research Output 2006 2019

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
Clustered Regularly Interspaced Short Palindromic Repeats
Huntington Disease
Trinucleotide Repeat Expansion
Neurodegenerative Diseases
Genes
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

REM Sleep
Eye Movements
Sleep
Neurons
Hypothalamus

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

Corpus Striatum
Pluripotent Stem Cells
Hydrogel
Huntington Disease
Stem cells

Innovations in CRISPR technology

Brooks, A. K. & Gaj, T., Aug 2018, In : Current Opinion in Biotechnology. 52, p. 95-101 7 p.

Research output: Contribution to journalReview article

Clustered Regularly Interspaced Short Palindromic Repeats
Innovation
Technology
Genes
Gene therapy

Manufacturing and delivering genome-editing proteins

Liu, J., Liang, Y. J., Ren, P. L. & Gaj, T., Jan 1 2018, Methods in Molecular Biology. Humana Press Inc., p. 253-273 21 p. (Methods in Molecular Biology; vol. 1867).

Research output: Chapter in Book/Report/Conference proceedingChapter

Ribonucleoproteins
Dependovirus
Zinc Fingers
Transgenes
Proteins

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

Clustered Regularly Interspaced Short Palindromic Repeats
barcoding
transform
Technology
cells
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

Pluripotent Stem Cells
Oligodendroglia
Stem cells
Cell culture
Clustered Regularly Interspaced Short Palindromic Repeats

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

Clustered Regularly Interspaced Short Palindromic Repeats
Amyotrophic Lateral Sclerosis
Motor Neurons
Spinal Cord
Dependovirus

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

Gene Knock-In Techniques
Dependovirus
Ribonucleoproteins
Guide RNA
Corpus Striatum
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

CRISPR-Cas Systems
Clustered Regularly Interspaced Short Palindromic Repeats
Dependovirus
Viruses
Genes

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

Dependovirus
Licensure
Neurons
Capsid
Neurosciences

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

Clustered Regularly Interspaced Short Palindromic Repeats
HIV-1
Viral Genes
Transcriptional Activation
HIV Infections

Genome-editing technologies: Principles and applications

Gaj, T., Sirk, S., Shui, S. L. & Liu, J., Jan 1 2016, In : Cold Spring Harbor Perspectives in Biology. 8, 12, a023754.

Research output: Contribution to journalArticle

Gene therapy
CRISPR-Associated Proteins
Genetic Therapy
Clustered Regularly Interspaced Short Palindromic Repeats
Genes

Genome engineering using Adeno-associated virus: Basic and clinical research applications

Gaj, T., Epstein, B. E. & Schaffer, D. V., Mar 1 2016, In : Molecular Therapy. 24, 3, p. 458-464 7 p.

Research output: Contribution to journalReview article

Dependovirus
Genome
Research
Gene Targeting
Homologous Recombination

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

Transcription
Human immunodeficiency virus 1
HIV-1
Transcription Factors
transcription factors
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

Zinc Fingers
Fingers
Zinc
Cells
Proteins

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

Stem cells
Stem Cells
Genes
Proteins
Zinc Fingers

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

Zinc Fingers
Genome
Nuclear Localization Signals
Proteins
Hematopoietic Stem Cells

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

Recombinases
zinc finger motif
Human Genome
Ports and harbors
Genes
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

Cell-Penetrating Peptides
nucleases
Transcription
engineering
Genes

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

Genes
Technology
Gene Editing
Editing

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

Recombinases
Dimers
targeting
genome
Genes

Expanding the scope of site-specific recombinases for genetic and metabolic engineering

Gaj, T., Sirk, S. & Barbas, C. F., Jan 1 2014, In : Biotechnology and bioengineering. 111, 1, p. 1-15 15 p.

Research output: Contribution to journalReview article

Metabolic engineering
Metabolic Engineering
Genetic engineering
Genetic Engineering
Genetic Recombination

Expanding the zinc-finger recombinase repertoire: Directed evolution and mutational analysis of serine recombinase specificity determinants

Sirk, S., 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

Recombinases
Zinc Fingers
Serine
DNA
Gene Rearrangement

Genome engineering with custom recombinases

Gaj, T. & Barbas, C. F., 2014, Methods in Enzymology. C ed. Academic Press Inc., p. 79-91 13 p. (Methods in Enzymology; vol. 546, no. C).

Research output: Chapter in Book/Report/Conference proceedingChapter

Recombinases
Genes
Genome
beta-Fructofuranosidase
Zinc Fingers

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

Zinc Fingers
Zinc
Proteins
Therapeutic Human Experimentation
Enzymes

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

Transcription factors
Transcription
Gene expression
Transcription Factors
Genes

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

Serum Albumin
Labeling
Lysine
Plasma (human)
Molecules

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

Zinc Fingers
Gene expression
Zinc
Genes
Genome
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

Recombinases
Zinc Fingers
Catalytic Domain
DNA
Synthetic Biology

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

Gaj, T., Gersbach, C. A. & Barbas, C. F., Jul 1 2013, In : Trends in Biotechnology. 31, 7, p. 397-405 9 p.

Research output: Contribution to journalReview article

Zinc Fingers
Transcription
Zinc
DNA
Genes
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

Recombinases
Zinc Fingers
DNA
Proteins
Catalytic DNA

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

Gene Knockout Techniques
Zinc Fingers
Zinc
Genes
Proteins
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

Recombinases
Serine
beta-Fructofuranosidase
Zinc Fingers
Mutagenesis

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

Recombinases
Serine
beta-Fructofuranosidase
Zinc Fingers
Mutagenesis

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

Recombinases
Zinc Fingers
Human Genome
Plasmids
Genome
2010

Directed evolution of an enhanced and highly efficient FokI cleavage domain for zinc finger nucleases

Guo, J., Gaj, T. & Barbas, C. F., Jul 1 2010, In : Journal of Molecular Biology. 400, 1, p. 96-107 12 p.

Research output: Contribution to journalArticle

Zinc Fingers
Mutagenesis
DNA Shuffling
Genetic Engineering
Genetic Therapy

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

Recombinases
Genes
Ampicillin Resistance
Human Genome
Substrate Specificity
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

Cyclic Peptides
Bacteriophages
Staurosporine
Cyclic AMP-Dependent Protein Kinases
Protein Kinases

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

Avidin
Recombinant Proteins
Immobilization
Purification
Peptides
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

Cyclic Peptides
Bacteriophages
Streptavidin
Avidin
Display devices