We have focused our efforts on diseases for which there are currently few or no available treatments because we want to maximize the impact of our technology for those who need it most.

Our research is still in the early stages. We hope you will use this website to learn more about genome editing, CRISPR and our areas of focus that might be useful. 

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Diverse Pipeline Across Range of Diseases

Program Pipeline Table

Systematic approach to demonstrating depth & breadth of genome editing platform

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Presentations & Publications

Publications and abstracts by Editas Medicine and foundational genome editing publications by our scientific founders.

Eric Tillotson, et al. An in vitro method for benchmarking of CRISPR-associated endonucleases. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017. Read More

Eugenio Marco, et al. UDiTasTM: A streamlined genome editing detection method for on- and off-target edits, large deletions, and translocations. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017. Read More

Maxwell N. Skor. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017. Read More

Cecilia Cotta-Ramusino. Characterization of Genomic Rearrangements in Response to CRISPR/Cas9-Induced Double-Stranded Breaks. Presented at Cold Spring Harbor Laboratory: Genome Engineering: The CRISPR-Cas9 Revolution, July 21-23, 2017. Read More

Barrett Steinberg, et al. Directed evolution platforms and applications for engineering RNA-guided nucleases. Presented at Synthetic Biology: Engineering Evolution & Design (SEED) 2017, Vancouver, BC, June 22, 2017. Read More

Hari Jayaram. Defining and characterizing the components of a CRISPR-Cas9 genomic medicine. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017. Read More

John Zuris, et al. Comparison of RNP-mediated editing by Type V Cpf1variants across multiple cell types. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017. Read More

Barrett Steinberg, et al. Directed evolution of Cas9 to reduce identified off-target cleavage. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017. Read More

Hari Jayaram, et al. Towards predictable editing: influence of target sequence, cell type, and choice of type II nuclease on desired repair outcomes. Presented at the CRISPR 2017 International Conference, Big Sky, Montana, June 8 – 10, 2017. Read More

Shannon Boye, Michael Stefanidakis, Rina Mepani, Maxwell Skor, Sebastian Gloskowski, Joy Horng, Kevin T. McCullough, Eugenio Marco, Georgia Giannoukos, Dawn Ciulla, Hoson Chao, George S. Bounoutas, C. Douglas Witherspoon, Christopher Wilson, David Bumcrot, Paul Gamlin, Sanford Boye, Haiyan Jiang, Charlie Albright, Morgan L. Maeder. Efficient In Vivo Gene Editing of Inherited Retinal Disease Genes in Mice and Non-Human Primates. Presented at the ASGCT 20th Annual Meeting, May 13, 2017. Read More

Georgia Giannoukos, Dawn Ciulla , Morgan Maeder, Sebastian Gloskowski, Max Skor, Vidya Dhanapal, Kiran Gogi, Fred Harbinski, Hayat Abdulkerim, Cecilia Cotta- Ramusino, Anne Bothmer, Eugenio Marco, Luis Barrera, Tongyao Wang, Hari Jayaram, Christopher Wilson, Vic Myer. UDiTaS(TM): A streamlined genome editing detection method for on- and off-target edits, large deletions, and translocations. Presented at the ASGCT 20th Annual Meeting, May 12, 2017. Read More

Carrie M. Margulies, Frank Buquicchio, Tanushree Phadke, John LaBella, Pietro Genovese, Giulia Schiroli, Valentina Vavassori, Charles Albright, Vic Myer, Luigi Naldini, Cecilia Cotta-Ramusino. Characterization of Targeted Integration with Viral and Non-Viral DNA Donors. Presented at the ASGCT 20th Annual Meeting, May 11, 2017. Read More

P. Singhal, A. Sadowski, R. Baral, M. Ahmed, M. Skor, G. S. Bounoutas, M. L. Maeder, M. Stefanidakis, V. Myer, A. E. Friedland. Self-inactivating Cas9: a method for reducing exposure while maintaining efficacy in virally-delivered Cas9 applications. Presented at the ASGCT 20th Annual Meeting, May 11, 2017. Read More

Jennifer Gori. CRISPR-mediated Editing of Hematopoietic Stem Cells for the Treatment of Beta-Hemoglobinopathies. Presented at the ASGCT 20th Annual Meeting, May 11, 2017. Read More

Shen Shen, Minerva E. Sanchez, Erik Corcoran, Keith Blomenkamp, Eugenio Marco, Jeffrey H. Teckman, David Bumcrot. Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice. Presented at the ASGCT 20th Annual Meeting, May 10, 2017. Read More

Ramya Viswanathan, et al. Comparison of RNP-mediated editing by Type V Cpf1 variants. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017. Read More

Guillaume Harmange, Eric Tillotson, William Selleck, Hari Jayaram, et al. Probing the Quality of Cas9 Ribonucleoprotein Complex Using Biochemistry and Biophysics. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017. Read More

Eric Tillotson, Guillaume Harmange, William Selleck, Hari Jayaram. Assessing Cas9-gRNA ribonucleoprotein complex formation for development of ex-vivo therapeutics. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017. Read More

Derek Cerchione, Barrett Steinberg, Morgan Maeder, Max Skor, Hari Jayaram, Vic Myer. Directed evolution of targeted Cas9 cleavage to the LCA10 splice donor mutation. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017. Read More

Haiyan Jiang. Opportunities and Challenges in Development of CRISPR Medicines. Presented at Broad Institute Genome Engineering 5.0, May 7-9, 2017. Read More

Cecilia Fernandez. Genome Editing: Considerations for Therapeutic Applications. Presented at the TIDES Oligonucleotide & Peptide Therapeutics conference, May 3, 2017. Read More

Christopher Wilson. Advancing CRISPR Technologies for Therapeutic Application. Presented at the TIDES Oligonucleotide & Peptide Therapeutics conference, May 3, 2017. Read More

Christopher Wilson, Georgia Giannoukos, Fred Harbinski, Michael Dinsmore, Dawn Ciulla, Vidya Dhanapal, Kiran Gogi, Greg Gotta, Eugenio Marco, Chris Borges, Luis Barrera, Tongyao Wang, Hari Jayaram, Sebastian Gloskowski, Morgan Maeder, Jack Heath, Jennifer Gori, Vic Myer. A Genome Editing Lead Finding Platform for Therapeutics. Cambridge, MA. Read More

Cecilia Cotta-Ramusino, et al. Effect of Different CRISPR/Cas9 Variants on Repair Pathway Choice. Presented at Keystone Symposia on Molecular and Cellular Biology: Genomic Instability and DNA Repair, April 2-6, 2017 Read More

Anne Bothmer, Hayat Abdulkerim, Ramya Viswanathan, John Zuris, Cecilia Cotta-Ramusino. DNA Repair Factor Overexpression Screen Identifies Factors Required for Repair Pathway Choice. Presented at Keystone Symposia on Molecular and Cellular Biology: Genomic Instability and DNA Repair, April 2-6, 2017 Read More

Anne Bothmer, Tanushree Phadke, Luis Barrera, Carrie Margulies, Hari Jayaram, Vic Meyer, Cecilia Cotta-Ramusino. Characterization of the Interplay Between DNA Repair and CRISPR/Cas9-Induced DNA Lesions. Presented at Keystone Symposia on Molecular and Cellular Biology: Precision Genome Engineering, January 8—12, 2017 Read More

Vic Myer. Advancing CRISPR Technologies for Therapeutic Application. Presented at the Keystone Genome Editing Meeting, January 5, 2017. Read More

J.M. Heath, A. Chalishazar, C.S. Lee, W. Selleck, C. Cotta-Ramusino, D. Bumcrot, J.L. Gori. Efficient CRISPR/Cas9 Mediated Gene Editing in Long-Term Engrafting Human Hematopoietic Stem Progenitor Cells. Presented at the American Society of Hematology Annual Meeting, December 3-6, 2016 in San Diego, CA. Read More

J.M Heath, A. Chalishazar, C.S. Lee, W. Selleck, C. Cotta-Ramusino, D. Bumcrot, J.L. Gori. AHighly Efficient CRISPR/Cas9 Mediated Gene Editing in Long-Term Engrafting Human Hematopoietic Stem Progenitor CellsA. Presented at the European Society of Gene and Cell Therapy Annual Meeting, October 18-21, 2016 in Florence, Italy. Read More

Morgan L. Maeder, Rina Mepani, Sebastian W. Gloskowski, Maxwell N. Skor, McKensie A. Collins, Gregory M. Gotta, Eugenio Marco, Luis A. Barrera, Hari Jayaram, David Bumcrot. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing. Presented at the Association for Research in Vision and Ophthalmology (ARVO), May 1-5, 2016 in Seattle, WA. Read More

J. M. Heath, A. Chalishazar, C.S. Lee, W. Selleck, C. Cotta-Ramusino, D. Bumcrot, J.L. Gori. Highly Efficient CRISPR/Cas9 Gene Editing and Long-Term Engraftment of Human Hematopoietic Stem and Progenitor Cells. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C. Read More

Morgan L. Maeder, Rina Mepani, Sebastian W. Gloskowski, Maxwell N. Skor, McKensie A. Collins, Gregory M. Gotta, Eugenio Marco, Luis A. Barrera, Hari Jayaram, David Bumcrot. Therapeutic Correction of an LCA-Causing Splice Defect in the CEP290 Gene by CRISPR/Cas-Mediated Genome Editing. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C. Read More

Josh Tycko, Nick Huston, Jacqueline N. Robinson-Hamm, Chris Wilson, Charles A. Gersbach, Patrick D. Hsu, David Bumcrot. Screening S. aureus CRISPR-Cas9 Paired Guide RNAs for Efficient Targeted Deletion in Duchenne Muscular Dystrophy. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C. Read More

Tanushree Phadke, Anne Bothmer, Christina Lee, Luis Barrera, Hari Jayaram, Vic Myer, Cecilia Cotta-Ramusino. DNA Ends Matter: The Impact of Using CRISPR/Cas9 Variants on DNA Repair Pathway Choice and Editing Profiles at The HBB Locus. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C. Read More

Grant Welstead, Justin Fang, Chris Nye, Frank Buquicchio, McKensie Collins, Will Selleck, Hari Jayaram, Ruth Salmon, Alex Baturevych, Sherri Mudri, Jon Jones, David Bumcrot, Blythe D. Sather. Successful Generation of CAR+PD-1- Primary T Cells Using Cas9-Mediated Genome Editing. Presented at the American Society of Gene and Cell Therapy Annual Meeting, May 4-7, 2016 in Washington, D.C. Read More

Morgan Maeder. Envisioning a Gene Editing Approach to Treat Inherited Blindness. Presented at the Federation of American Societies For Experimental Biology, June 5-10 in Lisbon, Portugal. Read More

David Bumcrot. Advancing CRISPR Medicines: Challenges and Solutions. Presented at the Oligonucleotide Therapeutics Society Annual Meeting, September 25-28 in Montreal, Canada.  Read More

C. Cotta-Ramusino, T. Phadke, M. Maeder, D. Bumcrot. Gene targeting of the HBB locus by Crispr/Cas9 to investigate repair pathway choice in response to different types of DNA lesions. Presented at Keystone Symposium for Genomic Instability and DNA Repair, March 2, 2015. Read More

J.L. Gori, G.G. Welstead, J. M. Heath, M.A. Collins, J.W. Fang, A.E. Friedland, D. Bumcrot. Cas9-mediated genome editing in hematopoietic stem/progenitor cells. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 13, 2015. Read More

William Selleck, A. Bothmer, C.Cotta, G. Welstead, D. Bumcrot and H. Jayaram. Biophysical Characterization and Direct Delivery of S. pyogenes Cas9 Ribonucleoprotein Complexes. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 13, 2015. Read More

G. Grant Welstead, Jennifer L. Gori, Justin Fang, McKensie Collins, Will Selleck, Ari Friedland, Hari Jayaram, David Bumcrot. Characterization of Cas9-mediated genome editing in human T cells. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 14, 2015. Read More

A.E. Friedland, S. Shen, P. Singhal, A. Sousa, M. Collins, M.L. Maeder, G.G. Welstead, H. Jayaram, D. Bumcrot. Staphyloccocus aureus Cas9 an alternative Cas9 for genome editing applications. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 15, 2015. Read More

C. Cotta-Ramusino, T. Phadke, M. Maeder, S. Moss, D. Bumcrot. Therapeutic Editing of the HBB Locus Using the Endogenous HBD Locus as a Donor Template. Presented at the 18th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 15, 2015. Read More

A.E. Friedland, R. Baral, P. Singhal, K. Loveluck, S. Shen, M. Sanchez, E. Marco, G. M. Gotta, M.L. Maeder, E.M. Kennedy, A.V.R. Kornepati, A. Sousa, M.A. Collins, H. Jayaram, B.R. Cullen, D. Bumcrot. Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications. Published in Genome Biology (2015) 16:257. Read More

A.E. Friedland, M.L. Maeder, H. Jayaram, G. Welstead, A. Sousa, S. Gloskowski, D. Bumcrot. Staphyloccocus aureus Cas9: An alternative Cas9 for genome editing applications. Presented at Keystone Symposium for Precision Genome Engineering and Synthetic Biology, January 12, 2015. Read More

Fu Y., Sander J.D., Reyon D., Cascio V.M., Joung K.J. Improving CRISPR-Cas nuclease specificity using truncated guide RNAs. Nature Biotechnology, 2014 Jan 26; doi: 10.1038/nbt2808. [Epub ahead of print] Read More

Sternberg, S.H., Redding, S., Jinek, M., Greene, E.C., Doudna, J.A. DNA interrogation by the CRISPR RNA-guided endonuclease Cas9. Nature (2014) doi:10.1038/nature13011. [Epub ahead of print] Read More

Jinek, M., Jiang, F.,Taylor, D.W., Sternberg, S.H., Kaya, E., Ma, E., Anders, C., Hauer, M., Zhou, K., Lin, S., Kaplan, M. Iavarone, A.T., Charpentier, E. Nogales, E., Doudna, J.A. Structures of Cas9 endonucleases reveal RNA-mediated conformational activation. Science, Feb. 6, 2014. DOI: 10.1126/science.1247997. [Epub ahead of print] Read More

Nishimasu, H., Ran, F.A., Hsu, P.D., Konermann, S., Shehata, S., Dohmae, N., Ishitani, R., Zhang, F. and Nureki, O. Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA. Cell, Feb. 13, 2014. DOI: 10.1016/j.cell.2014.02.001. [E-pub ahead of print] Read More

Sander, J.D. and Joung, J.K. CRISPR-Cas systems for editing, regulating and targeting genomes. Nature Biotechnology, March 2, 2014. doi:10.1038/ntb.2842. [E-pub ahead of print] Read More

Tsai, S.Q., Wyvekens, N., Khayter, C., Foden, J.A., Thapar, V., Reyon, D., Goodwin, M.J., Aryee, M.J., Joung, J.K. Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing. Nature Biotechnology, April 25, 2014. doi:10.1038/nbt.2908. [E-pub ahead of print] Read More

Guilinger, J.P., Thompson, D.B. and Liu, D.R. Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification. Nature Biotechnology, April 25, 2014. doi:10.1038/nbt.2909. [E-pub ahead of print] Read More

Hsu, P.D., Lander, E.S. and Zhang, F. Development and Applications of CRISPR-Cas9 for Genome Engineering. Cell, June 6, 2014. doi: http://dx.doi.org/10.1016/j.cell.2014.05.010. [E-pub ahead of print] Read More

Guell, M., Yang, L., and Church, G. Genome editing assessment using CRISPR Genome Analyzer (CRISPR-GA). Bioinformatics, July 1, 2014. doi: 10.1093/bioinformatics/btu427. [E-pub ahead of print] Read More

Sanjana, N.E., Shalem, O. and Zhang, F. Improved vectors and genome-wide libraries for CRISPR screening. Nature Methods, August 11, 2014. doi:10.1038/nmeth.3047. [E-pub ahead of print] Read More

O'Connell, M.R., Oakes, B.L., Stmberg, S.H., East-Seletsky, A., Kaplan, M., and Doudna, J.A. Programmable RNA recognition and cleavage by CRISPR/Cas9. Nature, September 28, 2014. doi: 10.1038/nature13769. [E-pub ahead of print] Read More

Platt, R.J., Chen,S., Zhou, Y., Yim, M.J., Swiech, L., Kempton, H.R., Dahlman, J.E., Parnas, O., Eisenhaure, T.M., Jovanovic, M., Graham, D.B., Jhunjhunwala, S., Heidenreich, M., Xavier, R.J., Langer, R., Anderson, D.G., Hacohen, N., Regev, A., Feng, G., Sharp, P.A., and Zhang, F. CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling. Cell, October 9, 2014. doi: http://dx.doi.org/10.1016/j.cell.2014.09.014. [E-pub ahead of print] Read More

Swiech, L., Heidenreich, M., Banerjee, A., Habib, N., Li, Y., Trombetta, J., Sur, M., and Zhang, F. In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9. Nature Biotechnology, October 19, 2014. doi: 10.1038/nbt.3055. [E-pub ahead of print] Read More

Zuris, J.A., Thompson, D.B., Shu, Y., Guilinger, J.P., Bessen, J.L., Hu, J.H., Maeder, M.L., Joung, J.K., Chen, Z.Y., and Liu, D.R. Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo. Nature Biotechnology, October 30, 2014. doi: 10.1038/nbt.3081. [E-pub ahead of print] Read More

Konermann, S., Brigham, M.D., Trevino, A.E., Joung, J., Abudayyeh, O.O., Barcena, C., Hsu, P.D., Habib, N., Gootenberg, J.S., Nishimasu, H., Nureki, O., Zhang, F. Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex. NCBI, December 10, 2014. doi:10.1038/nature14136. [Epub ahead of print] Read More

M. Jinek, A. East, A. Cheng, S. Lin, E. Ma, J. Doudna. RNA-programmed genome editing in human cells. Elife. 2013;2:e00471. Read More

J.K. Joung, J.D. Sander. TALENs: a widely applicable technology for targeted genome editing. Nature Reviews Molecular Cell Biology. 2013 Jan. doi:10.1038/nrm3486. Read More

L. Cong, F.A. Ran, D. Cox, S. Lin, R. Barretto, N. Habib, P.D. Hsu, X. Wu, W. Jiang, L.A. Marraffini, F. Zhang. Multiplex Genome Engineering Using CRISPR/Cas Systems. Science. 2013 Jan 3. doi:10.1126/science.1231143. Read More

P. Mali, L. Yang, K.M. Esvelt, J. Aach, M. Guell, J.E. DiCarlo, J.E. Norville, G.M. Church. RNA-Guided Human Genome Engineering via Cas9. Science. 2013 Jan 3. doi:10.1126/science.1232033. Read More

W. Jiang, D. Bikard, D. Cox, F. Zhang, L.A. Marraffini. RNA-guided editing of bacterial genomes using CRISPR-Cas systems. Nature Biotechnology. 2013 January 29. doi:10.1038/nbt.2508. Read More

W.Y. Hwang, Y. Fu, D. Reyon, M.L. Maeder, S.Q. Tsai, J.D. Sander, R.T. Peterson, J.J. Yeh, J.K. Joung. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nature Biotechnology. 2013 Jan 29. doi:10.1038/nbt.2501. Read More

L.S. Qi, M.H. Larson, L.A. Gilbert, J.A. Doudna, J.S. Weissman, A.P. Arkin, W.A. Lim. Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression. Cell. 28 February 2013. 10.1016/j.cell.2013.02.022. Read More

J.E. DiCarlo, J.E. Norville, P. Mali, X. Rios, J.Aach, G.M. Church. Genome engineering in Saccharomyces cerevisiae using CRISPR-Cas systems. Nucleic Acids Research. 2013 March 4. doi: 10.1093/nar/gkt135. Read More

E. Charpentier, J.A. Doudna. Biotechnology: Rewriting a genome. Nature. 2013 Mar 7;495(7439):50-1. Read More

M.L. Maeder, S.J. Linder, D. Reyon, J.F., Angstman, Y. Fu, J.D. Sander, J.K. Joung. Robust, synergistic regulation of human gene expression using TALE activators. Nat Methods. 2013 March 10. doi: 10.1038/nmeth.2366. Read More

H.Y. Lee, R.E. Haurwitz, A. Apffel, K. Zhou, B. Smart, C.D. Wenger, S. Laderman, L. Bruhn, J.A. Doudna. RNA-protein analysis using a conditional CRISPR nuclease. Proc Natl Acad Sci U S A. 2013 Apr 2;110(14):5416-21. Read More

M.J. Osborn, C.G. Starker, A.N. McElroy, B.R. Webber, M.J. Riddle, L. Xia, A.P. DeFeo, R. Gabriel, M. Schmidt, C.V. Kalle, D.F. Carlson, M.L. Maeder, J.K. Joung, J.E. Wagner, D.F. Voytas, B.R. Blazar, J.Tolar. TALEN-based Gene Correction for Epidermolysis Bullosa. Molecular Therapy. 2013 April 2. doi:10.1038/mt.2013.56. Read More

M.J. Osborn, C.G. Starker, A.N. McElroy, B.R. Webber, M.J. Riddle, L. Xia, A.P. DeFeo, R. Gabriel, M. Schmidt, C.V. Kalle, D.F. Carlson, M.L. Maeder, J.K. Joung, J.E. Wagner, D.F. Voytas, B.R. Blazar, J.Tolar. TALEN-based Gene Correction for Epidermolysis Bullosa. Molecular Therapy. 2013 April 2. doi:10.1038/mt.2013.56. Read More

D. Bikard, W. Jiang, P. Samai, A. Hochschild, F. Zhang, L.A. Marraffini. Programmable repression and activation of bacterial gene expression using an engineered CRISPR-Cas system. Nucleic Acids Research. 2013 June 12. doi: 10.1093/nar/gkt520. Read More

X. Li, E.R. Burnight, A.L. Cooney, N. Malani, T. Brady, J.D. Sander, J. Staber, S.J. Wheelan, J.K. Joung, P.B. Jr. McCray, F.D. Bushman, P.L. Sinn, N.L. Craig. piggyBac transposase tools for genome engineering. Proc Natl Acad Sci U S A. 2013 Jun 18;110(25):E2279-87. Read More

Y. Fu, J.A. Foden, C. Khayter, M.L. Maeder, D. Reyon, J.K. Joung, J.D. Sander. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nature Biotechnology. 2013 June 23. doi:10.1038/nbt.2623. Read More

A.E. Friedland, Y.B. Tzur, K.M. Esvelt, M.P. Colaiácovo, G.M. Church, J.A. Calarco. Heritable genome editing in C. elegans via a CRISPR-Cas9 system. Nature Methods. 2013 June 30. doi:10.1038/nmeth.2532. Read More

D. Reyon, M.L. Maeder, C. Khayter, S.Q. Tsai, J.E. Foley, J.D. Sander, J.K. Joung. Engineering Customized TALE Nucleases (TALENs) and TALE Transcription Factors by Fast Ligation-Based Automatable Solid-Phase High-Throughput (FLASH) Assembly. Current Protocols in Molecular Biology. 2013 July 1. doi:10.1002/0471142727.mb1216s103. Read More

W.Y. Hwang, Y. Fu, D. Reyon, M.L. Maeder, P. Kaini, J.D. Sander, J.K. Joung, R.T. Peterson, J.J. Yeh. Heritable and Precise Zebrafish Genome Editing Using a CRISPR-Cas System. PLOS One. 2013 July 9. doi: 10.1371/journal.pone.0068708. Read More

L.A. Gilbert, M. H. Larson, L. Morsut, Z. Liu, G.A. Brar, S.E. Torres, N. Stern-Ginossar, O. Brandman, E.H. Whitehead, J.A. Doudna, W.A. Lim, J.S. Weissman, L.S. Qi. CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes. Cell. 2013 July 11.10.1016/j.cell.2013.06.044. Read More

P.D. Hsu, D.A. Scott, J.A. Weinstein, F.A. Ran, S. Konermann, V. Agarwala, Y. Li, E.J. Fine, X. Wu, O. Shalem, T.J. Cradick, L.A. Marraffini, G. Bao, F. Zhang. DNA targeting specificity of RNA-guided Cas9 nucleases. Nature Biotechnology. 2013 July 21. doi:10.1038/nbt.2647. Read More

S. Konermann, M.D. Brigham, A.E. Trevino, P.D. Hsu, M. Heidenreich, L. Cong, R.J. Platt, D.A. Scott, G.M. Church, F. Zhang. Optical control of mammalian endogenous transcription and epigenetic states. Nature. 2013 July 23. doi:10.1038/nature12466. Read More

M.L. Maeder, S.J. Linder, V.M. Cascio, Y. Fu, Q.H. Ho, J.K. Joung. CRISPR RNA–guided activation of endogenous human genes. Nature Methods. 2013 July 25. doi:10.1038/nmeth.2598. Read More

L. Yang, M. Guell, S. Byrne, J.L. Yang, A. De Los Angeles, P. Mali, J. Aach, C. Kim-Kiselak, A.W. Briggs, X. Rios, P. Huang, G. Daley, G. Church. Optimization of scarless human stem cell genome editing. Nucleic Acids Research. 2013 July 31. doi: 10.1093/nar/gkt555. Read More

P. Mali, J. Aach, P.B. Stranges, K.M. Esvelt, M. Moosburner, S. Kosuri, L. Yang, G.M. Church. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nature Biotechnology. 2013 Aug 1. doi:10.1038/nbt.2675. Read More

J. Li, J.E. Norville, J. Aach, M. McCormack, D. Zhang, J. Bush, G.M. Church, J. Sheen. Multiplex and homologous recombination–mediated genome editing in Arabidopsis and Nicotiana benthamiana using guide RNA and Cas9. Nature Biotechnology. 2013 Aug 8. doi:10.1038/nbt.2654. Read More

T. Lo, C.S. Pickle, S. Lin, E.J. Ralston, M. Gurling, C.M. Schartner, Q. Bian, J.A. Doudna, B.J. Meyer. Precise and Heritable Genome Editing in Evolutionarily Diverse Nematodes Using TALENs and CRISPR/Cas9 to Engineer Insertions and Deletions. Genetics. Advance online publication. 2013 Aug 9. doi:10.1534/genetics.113.155382. Read More

V. Pattanayak, S. Lin, J.P. Guilinger, E. Ma, J.A. Doudna, D.R. Liu. High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity. Nature Biotechnology. 2013 Aug 11. doi:10.1038/nbt.2673. Read More

J.D. Sander, C.L. Ramirez, S.J. Linder, V. Pattanayak, N. Shoresh, M.Ku, J.A. Foden, D. Reyon, B.E. Bernstein, D.R. Liu, J.K. Joung. In silico abstraction of zinc finger nuclease cleavage profiles reveals an expanded landscape of off-target sites. Nucleic Acids Research. 2013 Aug 14. doi:10.1093/nar/gkt716. Read More

Z. Feng, B. Zhang, W. Ding, X. Liu, D. Yang, P. Wei, F. Cao, S. Zhu, F. Zhang, Y. Mao, J. Zhu. Efficient genome editing in plants using a CRISPR/Cas system. Cell Research. 2013 Aug 20. doi:10.1038/cr.2013.114. Read More

Y.B. Tzur, A.E. Friedland, S. Nadarajan, G.M. Church, J.A. Calarco, M.P. Colaiácovo. Heritable Custom Genomic Modifications in Caenorhabditis elegans via a CRISPR–Cas9 System. Genetics. Advance online publication. 2013 Aug 26. doi:10.1534/genetics.113.156075. Read More

F.A. Ran, P.D. Hsu, C. Lin, J.S. Gootenberg, S. Konermann, A.E. Trevino, D.A. Scott, A. Inoue, S. Matoba, Y. Zhang, F. Zhang. Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity. Cell. 2013 Aug 29. doi:10.1016/j.cell.2013.08.021. Read More

E.M. Mendenhall, K.E. Williamson, D. Reyon, J.Y. Zou, O. Ram, J.K. Joung, B.E. Bernstein. Locus-specific editing of histone modifications at endogenous enhancers. Nat Biotechnol. 2013 Dec;31(12):1133-6. doi: 10.1038/nbt.2701. Epub 2013 Sep 8. Read More

P. Mali, K.M. Esvelt, G.M. Church. Cas9 as a versatile tool for engineering biology. Nat Methods. 2013 Oct;10(10):957-63. Read More

Y. Qi, X. Li, Y. Zhang, C.G. Starker, N.J. Baltes, F. Zhang, J.D. Sander, D. Reyon, J.K. Joung, D.F. Voytas. Targeted deletion and inversion of tandemly arrayed genes in Arabidopsis thaliana using zinc finger nucleases. G3 (Bethesda). 2013 Oct 3;3(10):1707-15. Read More

O. Niewoehner, M. Jinek, J.A. Doudna. Evolution of CRISPR RNA recognition and processing by Cas6 endonucleases. Nucleic Acids Res. 2013 Oct 22. Read More

K.M. Esvelt, P. Mali, J.L. Braff, M. Moosburner, S.J. Yaung, G.M. Church. Orthogonal Cas9 proteins for RNA-guided gene regulation and editing. Nat Methods. 2013 Nov;10(11):1116-21. Read More

O. Shalem, N.E. Sanjana, E. Hartenian, X. Shi, D.A. Scott, T. Mikkelson, D. Heckl, B.L. Ebert, D.E. Root, J.G. Doench, F. Zhang. Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells. Science. doi: 10.1126/science.1247005. Epub 2013 Dec 12. Read More

M.L. Maeder, J.F. Angstman, M.E. Richardson, S.J. Linder, V.M. Cascio, S.Q. Tsai, Q.H. Ho, J.D. Sander, D. Reyon, B.E. Bernstein, J.F. Costello, M.F. Wilkinson, J.K. Joung. Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins. Nat Biotechnol. 2013 Dec;31(12):1137-42. doi: 10.1038/nbt.2726. Epub 2013 Oct 9. Read More

M. Jinek, K. Chylinski, I. Fonfara, M. Hauer, J.A. Doudna, E. Charpentier. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science. 2012 Aug 17;337(6096):816-21. doi: 10.1126/science.1225829. Epub 2012 Jun 28. Read More

C.L. Ramirez, M.T. Certo, C. Mussolino, M.J. Goodwin, T.J. Cradick, A.P. McCaffrey, T. Cathomen, A.M. Scharenberg, J.K. Joung. Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects. Nucleic Acids Res. 2012 Jul;40(12):5560-8. Read More

A.S. Khalil, T.K. Lu, C.J. Bashor, C.L. Ramirez, N.C. Pyenson, J.K. Joung, J.J. Collins. A synthetic biology framework for programming eukaryotic transcription functions. Cell. 2012 Aug 3;150(3):647-58. Read More

D. Reyon, C. Khayter, M.R. Regan, J.K. Joung, J.D. Sander. Engineering Designer Transcription Activator--Like Effector Nucleases (TALENs) by REAL or REAL-Fast Assembly. Current Protocols in Molecular Biology. 2012 Oct 1. doi: 10.1002/0471142727.mb1215s100. Read More