Generation of knock-in primary human T cells using Cas9 ribonucleoproteins
Abstract
T-cell genome engineering holds great promise for cell-based therapies for cancer, HIV, primary immune deficiencies, and autoimmune diseases, but genetic manipulation of human T cells has been challenging. Improved tools are needed to efficiently “knock out” genes and “knock in” targeted genome modifications to modulate T-cell function and correct disease-associated mutations. CRISPR/Cas9 technology is facilitating genome engineering in many cell types, but in human T cells its efficiency has been limited and it has not yet proven useful for targeted nucleotide replacements. Here we report efficient genome engineering in human CD4+ T cells using Cas9:single-guide RNA ribonucleoproteins (Cas9 RNPs). Cas9 RNPs allowed ablation of CXCR4, a coreceptor for HIV entry. Cas9 RNP electroporation caused up to ~40% of cells to lose high-level cell-surface expression of CXCR4, and edited cells could be enriched by sorting based on low CXCR4 expression. Importantly, Cas9 RNPs paired with homology-directed repair template oligonucleotides generated a high frequency of targeted genome modifications in primary T cells. Targeted nucleotide replacement was achieved in CXCR4 and PD-1 (PDCD1), a regulator of T-cell exhaustion that is a validated target for tumor immunotherapy. Deep sequencing of a target site confirmed that Cas9 RNPs generated knock-in genome modifications with up tomore »
- Authors:
-
- Univ. of California, San Francisco, CA (United States)
- Univ. of California, Berkeley, CA (United States)
- Univ. of California, San Francisco, CA (United States); Univ. of California, Berkeley, CA (United States); Lawrence Berkeley National Lab. (LBNL), Berkeley, CA (United States)
- Univ. of California, San Francisco, CA (United States); Univ. of California, Berkeley, CA (United States)
- Publication Date:
- Research Org.:
- Lawrence Berkeley National Laboratory (LBNL), Berkeley, CA (United States)
- Sponsoring Org.:
- USDOE
- OSTI Identifier:
- 1221821
- Grant/Contract Number:
- AC02-05CH11231
- Resource Type:
- Accepted Manuscript
- Journal Name:
- Proceedings of the National Academy of Sciences of the United States of America
- Additional Journal Information:
- Journal Volume: 112; Journal Issue: 33; Journal ID: ISSN 0027-8424
- Publisher:
- National Academy of Sciences, Washington, DC (United States)
- Country of Publication:
- United States
- Language:
- English
- Subject:
- 60 APPLIED LIFE SCIENCES; CRISPR/Cas9; genome engineering; Cas9 ribonucleoprotein; RNP primary; human T cells
Citation Formats
Schumann, Kathrin, Lin, Steven, Boyer, Eric, Simeonov, Dimitre R., Subramaniam, Meena, Gate, Rachel E., Haliburton, Genevieve E., Ye, Chun J., Bluestone, Jeffrey A., Doudna, Jennifer A., and Marson, Alexander. Generation of knock-in primary human T cells using Cas9 ribonucleoproteins. United States: N. p., 2015.
Web. doi:10.1073/pnas.1512503112.
Schumann, Kathrin, Lin, Steven, Boyer, Eric, Simeonov, Dimitre R., Subramaniam, Meena, Gate, Rachel E., Haliburton, Genevieve E., Ye, Chun J., Bluestone, Jeffrey A., Doudna, Jennifer A., & Marson, Alexander. Generation of knock-in primary human T cells using Cas9 ribonucleoproteins. United States. https://doi.org/10.1073/pnas.1512503112
Schumann, Kathrin, Lin, Steven, Boyer, Eric, Simeonov, Dimitre R., Subramaniam, Meena, Gate, Rachel E., Haliburton, Genevieve E., Ye, Chun J., Bluestone, Jeffrey A., Doudna, Jennifer A., and Marson, Alexander. Mon .
"Generation of knock-in primary human T cells using Cas9 ribonucleoproteins". United States. https://doi.org/10.1073/pnas.1512503112. https://www.osti.gov/servlets/purl/1221821.
@article{osti_1221821,
title = {Generation of knock-in primary human T cells using Cas9 ribonucleoproteins},
author = {Schumann, Kathrin and Lin, Steven and Boyer, Eric and Simeonov, Dimitre R. and Subramaniam, Meena and Gate, Rachel E. and Haliburton, Genevieve E. and Ye, Chun J. and Bluestone, Jeffrey A. and Doudna, Jennifer A. and Marson, Alexander},
abstractNote = {T-cell genome engineering holds great promise for cell-based therapies for cancer, HIV, primary immune deficiencies, and autoimmune diseases, but genetic manipulation of human T cells has been challenging. Improved tools are needed to efficiently “knock out” genes and “knock in” targeted genome modifications to modulate T-cell function and correct disease-associated mutations. CRISPR/Cas9 technology is facilitating genome engineering in many cell types, but in human T cells its efficiency has been limited and it has not yet proven useful for targeted nucleotide replacements. Here we report efficient genome engineering in human CD4+ T cells using Cas9:single-guide RNA ribonucleoproteins (Cas9 RNPs). Cas9 RNPs allowed ablation of CXCR4, a coreceptor for HIV entry. Cas9 RNP electroporation caused up to ~40% of cells to lose high-level cell-surface expression of CXCR4, and edited cells could be enriched by sorting based on low CXCR4 expression. Importantly, Cas9 RNPs paired with homology-directed repair template oligonucleotides generated a high frequency of targeted genome modifications in primary T cells. Targeted nucleotide replacement was achieved in CXCR4 and PD-1 (PDCD1), a regulator of T-cell exhaustion that is a validated target for tumor immunotherapy. Deep sequencing of a target site confirmed that Cas9 RNPs generated knock-in genome modifications with up to ~20% efficiency, which accounted for up to approximately one-third of total editing events. These results establish Cas9 RNP technology for diverse experimental and therapeutic genome engineering applications in primary human T cells.},
doi = {10.1073/pnas.1512503112},
journal = {Proceedings of the National Academy of Sciences of the United States of America},
number = 33,
volume = 112,
place = {United States},
year = {Mon Jul 27 00:00:00 EDT 2015},
month = {Mon Jul 27 00:00:00 EDT 2015}
}
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Works referenced in this record:
Adoptive Immunotherapy for Cancer or Viruses
journal, March 2014
- Maus, Marcela V.; Fraietta, Joseph A.; Levine, Bruce L.
- Annual Review of Immunology, Vol. 32, Issue 1
Helper T-cell identity and evolution of differential transcriptomes and epigenomes
journal, February 2013
- Vahedi, Golnaz; C. Poholek, Amanda; Hand, Timothy W.
- Immunological Reviews, Vol. 252, Issue 1
HIV-1 Entry Cofactor: Functional cDNA Cloning of a Seven-Transmembrane, G Protein-Coupled Receptor
journal, May 1996
- Feng, Y.; Broder, C. C.; Kennedy, P. E.
- Science, Vol. 272, Issue 5263
Chimeric Antigen Receptor–Modified T Cells in Chronic Lymphoid Leukemia
journal, August 2011
- Porter, David L.; Levine, Bruce L.; Kalos, Michael
- New England Journal of Medicine, Vol. 365, Issue 8
Adoptive immunotherapy for cancer: harnessing the T cell response
journal, March 2012
- Restifo, Nicholas P.; Dudley, Mark E.; Rosenberg, Steven A.
- Nature Reviews Immunology, Vol. 12, Issue 4
Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9
journal, November 2014
- Mandal, Pankaj K.; Ferreira, Leonardo M. R.; Collins, Ryan
- Cell Stem Cell, Vol. 15, Issue 5
Multifactorial T-cell Hypofunction That Is Reversible Can Limit the Efficacy of Chimeric Antigen Receptor-Transduced Human T cells in Solid Tumors
journal, June 2014
- Moon, E. K.; Wang, L. -C.; Dolfi, D. V.
- Clinical Cancer Research, Vol. 20, Issue 16
Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases
journal, November 2013
- Sung, Y. H.; Kim, J. M.; Kim, H. -T.
- Genome Research, Vol. 24, Issue 1
Targeted genome editing in human repopulating haematopoietic stem cells
journal, May 2014
- Genovese, Pietro; Schiroli, Giulia; Escobar, Giulia
- Nature, Vol. 510, Issue 7504
Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
journal, March 2014
- Tebas, Pablo; Stein, David; Tang, Winson W.
- New England Journal of Medicine, Vol. 370, Issue 10
Long-Term Control of HIV by CCR5 Delta32/Delta32 Stem-Cell Transplantation
journal, February 2009
- Hütter, Gero; Nowak, Daniel; Mossner, Maximilian
- New England Journal of Medicine, Vol. 360, Issue 7
Genetic and epigenetic fine mapping of causal autoimmune disease variants
journal, October 2014
- Farh, Kyle Kai-How; Marson, Alexander; Zhu, Jiang
- Nature, Vol. 518, Issue 7539
CD4+ T Cells from IPEX Patients Convert into Functional and Stable Regulatory T Cells by FOXP3 Gene Transfer
journal, December 2013
- Passerini, L.; Mel, E. R.; Sartirana, C.
- Science Translational Medicine, Vol. 5, Issue 215
Anti-PD-1 Antibody Therapy Potently Enhances the Eradication of Established Tumors By Gene-Modified T Cells
journal, July 2013
- John, L. B.; Devaud, C.; Duong, C. P. M.
- Clinical Cancer Research, Vol. 19, Issue 20
The new frontier of genome engineering with CRISPR-Cas9
journal, November 2014
- Doudna, Jennifer A.; Charpentier, Emmanuelle
- Science, Vol. 346, Issue 6213
Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery
journal, December 2014
- Lin, Steven; Staahl, Brett T.; Alla, Ravi K.
- eLife, Vol. 3
Immune Checkpoint Blockade: A Common Denominator Approach to Cancer Therapy
journal, April 2015
- Topalian, Suzanne L.; Drake, Charles G.; Pardoll, Drew M.
- Cancer Cell, Vol. 27, Issue 4
Development and Applications of CRISPR-Cas9 for Genome Engineering
journal, June 2014
- Hsu, Patrick D.; Lander, Eric S.; Zhang, Feng
- Cell, Vol. 157, Issue 6
Function of the chemokine receptor CXCR4 in haematopoiesis and in cerebellar development
journal, June 1998
- Zou, Yong-Rui; Kottmann, Andreas H.; Kuroda, Masahiko
- Nature, Vol. 393, Issue 6685
Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins
journal, April 2014
- Kim, S.; Kim, D.; Cho, S. W.
- Genome Research, Vol. 24, Issue 6
Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
journal, October 2014
- Zuris, John A.; Thompson, David B.; Shu, Yilai
- Nature Biotechnology, Vol. 33, Issue 1
Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection
journal, January 2014
- Didigu, Chuka A.; Wilen, Craig B.; Wang, Jianbin
- Blood, Vol. 123, Issue 1
Double-Strand Break End Resection and Repair Pathway Choice
journal, December 2011
- Symington, Lorraine S.; Gautier, Jean
- Annual Review of Genetics, Vol. 45, Issue 1
A seven-transmembrane domain receptor involved in fusion and entry of T-cell-tropic human immunodeficiency virus type 1 strains.
journal, January 1996
- Berson, J. F.; Long, D.; Doranz, B. J.
- Journal of virology, Vol. 70, Issue 9
Immune Checkpoint Blockade: A Common Denominator Approach to Cancer Therapy
journal, April 2015
- Topalian, Suzanne L.; Drake, Charles G.; Pardoll, Drew M.
- Cancer Cell, Vol. 27, Issue 4
Helper T-cell identity and evolution of differential transcriptomes and epigenomes
journal, February 2013
- Vahedi, Golnaz; C. Poholek, Amanda; Hand, Timothy W.
- Immunological Reviews, Vol. 252, Issue 1
Double-Strand Break End Resection and Repair Pathway Choice
journal, December 2011
- Symington, Lorraine S.; Gautier, Jean
- Annual Review of Genetics, Vol. 45, Issue 1
Works referencing / citing this record:
Acoustophoretic rapid media exchange and continuous-flow electrotransfection of primary human T cells for applications in automated cellular therapy manufacturing
journal, January 2019
- Hsi, Peter; Christianson, Rebecca J.; Dubay, Ryan A.
- Lab on a Chip, Vol. 19, Issue 18
The next generation of CRISPR–Cas technologies and applications
journal, May 2019
- Pickar-Oliver, Adrian; Gersbach, Charles A.
- Nature Reviews Molecular Cell Biology, Vol. 20, Issue 8
Efficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage
journal, February 2017
- Zhang, Jian-Ping; Li, Xiao-Lan; Li, Guo-Hua
- Genome Biology, Vol. 18, Issue 1
Genome editing by natural and engineered CRISPR-associated nucleases
journal, June 2018
- Wu, Wen Y.; Lebbink, Joyce H. G.; Kanaar, Roland
- Nature Chemical Biology, Vol. 14, Issue 7
CRISPR/Cas9 genome editing in human hematopoietic stem cells
journal, January 2018
- Bak, Rasmus O.; Dever, Daniel P.; Porteus, Matthew H.
- Nature Protocols, Vol. 13, Issue 2
Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy
journal, December 2018
- Wang, Le; Zheng, Wenfu; Liu, Shaoqin
- ChemBioChem
Genome editing of bread wheat using biolistic delivery of CRISPR/Cas9 in vitro transcripts or ribonucleoproteins
journal, February 2018
- Liang, Zhen; Chen, Kunling; Zhang, Yi
- Nature Protocols, Vol. 13, Issue 3
Advancing chimeric antigen receptor T cell therapy with CRISPR/Cas9
journal, April 2017
- Ren, Jiangtao; Zhao, Yangbing
- Protein & Cell, Vol. 8, Issue 9
Orthotopic replacement of T-cell receptor α- and β-chains with preservation of near-physiological T-cell function
journal, June 2019
- Schober, Kilian; Müller, Thomas R.; Gökmen, Füsun
- Nature Biomedical Engineering, Vol. 3, Issue 12
Helios enhances the preferential differentiation of human fetal CD4 + naïve T cells into regulatory T cells
journal, November 2019
- Ng, Melissa S. F.; Roth, Theodore L.; Mendoza, Ventura F.
- Science Immunology, Vol. 4, Issue 41
Application of Genome Editing Techniques in Immunology
journal, January 2018
- Zych, Agata O.; Bajor, Malgorzata; Zagozdzon, Radoslaw
- Archivum Immunologiae et Therapiae Experimentalis, Vol. 66, Issue 4
Therapeutic applications of CRISPR RNA-guided genome editing
journal, August 2016
- Koo, Taeyoung; Kim, Jin-Soo
- Briefings in Functional Genomics, Vol. 16, Issue 1
Strategies for nonviral nanoparticle‐based delivery of CRISPR/Cas9 therapeutics
journal, December 2019
- Chen, Fengqian; Alphonse, Martin; Liu, Qi
- WIREs Nanomedicine and Nanobiotechnology
The human leukemia virus HTLV-1 alters the structure and transcription of host chromatin in cis
journal, March 2018
- Melamed, Anat; Yaguchi, Hiroko; Miura, Michi
- eLife
CRISPR/Cas9-Mediated Genome Editing in Epstein-Barr Virus-Transformed Lymphoblastoid B-Cell Lines: CRISPR in EBV-Transformed B-Cells
journal, January 2018
- Jiang, Sizun; Wang, Liang Wei; Walsh, Michael J.
- Current Protocols in Molecular Biology, Vol. 121, Issue 1
Emerging CRISPR/Cas9 applications for T-cell gene editing
journal, April 2019
- Preece, Roland; Georgiadis, Christos
- Emerging Topics in Life Sciences, Vol. 3, Issue 3
CRISPR/Cas9 Genome Editing Using Gold-Nanoparticle-Mediated Laserporation
journal, May 2018
- Bošnjak, Berislav; Permanyer, Marc; Sethi, Maya K.
- Advanced Biosystems, Vol. 2, Issue 11
Metabolic reprogramming of human CD8 + memory T cells through loss of SIRT1
journal, November 2017
- Jeng, Mark Y.; Hull, Philip A.; Fei, Mingjian
- The Journal of Experimental Medicine, Vol. 215, Issue 1
Functional Interrogation of Primary Human T Cells via CRISPR Genetic Editing
journal, July 2018
- Chen, Xin; Kozhaya, Lina; Tastan, Cihan
- The Journal of Immunology, Vol. 201, Issue 5
Delivery technologies for genome editing
journal, March 2017
- Yin, Hao; Kauffman, Kevin J.; Anderson, Daniel G.
- Nature Reviews Drug Discovery, Vol. 16, Issue 6
An efficient electroporation protocol for the genetic modification of mammalian cells
journal, September 2016
- Chicaybam, Leonardo; Barcelos, Camila; Peixoto, Barbara
- Frontiers in Bioengineering and Biotechnology
CRISPR-delivery particles targeting nuclear receptor–interacting protein 1 ( Nrip1 ) in adipose cells to enhance energy expenditure
journal, September 2018
- Shen, Yuefei; Cohen, Jessica L.; Nicoloro, Sarah M.
- Journal of Biological Chemistry, Vol. 293, Issue 44
Transfection by Electroporation
journal, January 2018
- Potter, Huntington; Heller, Richard
- Current Protocols in Molecular Biology, Vol. 121, Issue 1
Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing
journal, November 2017
- Yin, Hao; Song, Chun-Qing; Suresh, Sneha
- Nature Biotechnology, Vol. 35, Issue 12
Recent advances in DNA-free editing and precise base editing in plants
journal, November 2017
- Zhang, Yi; Gao, Caixia
- Emerging Topics in Life Sciences, Vol. 1, Issue 2
Genome-wide CRISPR Screens in Primary Human T Cells Reveal Key Regulators of Immune Function
journal, December 2018
- Shifrut, Eric; Carnevale, Julia; Tobin, Victoria
- Cell, Vol. 175, Issue 7
Delivering CRISPR: a review of the challenges and approaches
journal, January 2018
- Lino, Christopher A.; Harper, Jason C.; Carney, James P.
- Drug Delivery, Vol. 25, Issue 1
Highly efficient and versatile plasmid-based gene editing in primary T cells
posted_content, January 2018
- Kornete, Mara; Marone, Romina; Jeker, Lukas T.
- The Journal of Immunology
Resources for the design of CRISPR gene editing experiments
journal, November 2015
- Graham, Daniel B.; Root, David E.
- Genome Biology, Vol. 16, Issue 1
Viable and efficient electroporation-based genetic manipulation of unstimulated human T cells
posted_content, October 2019
- Aksoy, Pinar; Aksoy, Bülent Arman; Czech, Eric
- BioRxiv
Cancer diagnosis and immunotherapy in the age of CRISPR
journal, December 2018
- Cook, Peter J.; Ventura, Andrea
- Genes, Chromosomes and Cancer, Vol. 58, Issue 4
CRISPR/Cas9: A tool for immunological research
journal, February 2018
- Hochheiser, Katharina; Kueh, Andrew J.; Gebhardt, Thomas
- European Journal of Immunology, Vol. 48, Issue 4
Recent challenges and advances in genetically-engineered cell therapy
journal, December 2017
- Yong, Seok-Beom; Chung, Jee Young; Song, Yoonsung
- Journal of Pharmaceutical Investigation, Vol. 48, Issue 2
CRISPR/Cas9 technology as a potent molecular tool for gene therapy
journal, December 2018
- Karimian, Ansar; Azizian, Khalil; Parsian, Hadi
- Journal of Cellular Physiology, Vol. 234, Issue 8
An efficient and scalable pipeline for epitope tagging in mammalian stem cells using Cas9 ribonucleoprotein
journal, January 2018
- Dewari, Pooran Singh; Southgate, Benjamin; Mccarten, Katrina
- eLife
CRISPR/Cas-mediated knock-in via non-homologous end-joining in the crustacean Daphnia magna
journal, October 2017
- Kumagai, Hitoshi; Nakanishi, Takashi; Matsuura, Tomoaki
- PLOS ONE, Vol. 12, Issue 10
CRISPR–Cas9 genome engineering of primary CD4+ T cells for the interrogation of HIV–host factor interactions
journal, December 2018
- Hultquist, Judd F.; Hiatt, Joseph; Schumann, Kathrin
- Nature Protocols, Vol. 14, Issue 1
HIV-1 inhibition in cells with CXCR4 mutant genome created by CRISPR-Cas9 and piggyBac recombinant technologies
journal, June 2018
- Liu, Shuai; Wang, Qiankun; Yu, Xiao
- Scientific Reports, Vol. 8, Issue 1
Engineered T cells: the promise and challenges of cancer immunotherapy
journal, August 2016
- Fesnak, Andrew D.; June, Carl H.; Levine, Bruce L.
- Nature Reviews Cancer, Vol. 16, Issue 9
CRISPR-Cas9-mediated multiplex gene editing in CAR-T cells
journal, December 2016
- Liu, Xiaojuan; Zhang, Yongping; Cheng, Chen
- Cell Research, Vol. 27, Issue 1
Resolving mechanisms of immune-mediated disease in primary CD4 T cells.
text, January 2020
- Bourges, Christophe; Groff, Abigail F.; Burren, Oliver
- Apollo - University of Cambridge Repository
In vitro–transcribed guide RNAs trigger an innate immune response via the RIG-I pathway
journal, July 2018
- Wienert, Beeke; Shin, Jiyung; Zelin, Elena
- PLOS Biology, Vol. 16, Issue 7
Applications of CRISPR systems in respiratory health: Entering a new ‘red pen’ era in genome editing
journal, February 2019
- Moses, Colette; Kaur, Parwinder
- Respirology, Vol. 24, Issue 7
Genome-wide CRISPR Screens in Primary Human T Cells Reveal Key Regulators of Immune Function
journal, August 2018
- Shifrut, Eric; Carnevale, Julia; Tobin, Victoria
- Cell
Primary allogeneic mitochondrial mix (PAMM) transfer/transplant by MitoCeption to address damage in PBMCs caused by ultraviolet radiation
journal, June 2019
- Cabrera, Francisco; Ortega, Mayra; Velarde, Francesca
- BMC Biotechnology, Vol. 19, Issue 1
The clinical potential of gene editing as a tool to engineer cell‐based therapeutics
journal, January 2020
- Ashmore‐Harris, Candice; Fruhwirth, Gilbert O.
- Clinical and Translational Medicine, Vol. 9, Issue 1
Genetic abrogation of immune checkpoints in antigen-specific cytotoxic T-lymphocyte as a potential alternative to blockade immunotherapy
journal, April 2018
- Zhang, Chi; Peng, Yanchun; Hublitz, Philip
- Scientific Reports, Vol. 8, Issue 1
Carboxylated branched poly(β-amino ester) nanoparticles enable robust cytosolic protein delivery and CRISPR-Cas9 gene editing
journal, December 2019
- Rui, Yuan; Wilson, David R.; Choi, John
- Science Advances, Vol. 5, Issue 12
Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
journal, October 2016
- DeWitt, M. A.; Magis, W.; Bray, N. L.
- Science Translational Medicine, Vol. 8, Issue 360
GapmeR cellular internalization by macropinocytosis induces sequence-specific gene silencing in human primary T-cells
journal, November 2016
- Fazil, Mobashar Hussain Urf Turabe; Ong, Seow Theng; Chalasani, Madhavi Latha Somaraju
- Scientific Reports, Vol. 6, Issue 1
Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome
journal, May 2016
- Hubbard, Nicholas; Hagin, David; Sommer, Karen
- Blood, Vol. 127, Issue 21
Epigenetic changes around the pX region and spontaneous HTLV-1 transcription are CTCF-independent
journal, January 2018
- Miura, Michi; Miyazato, Paola; Satou, Yorifumi
- Wellcome Open Research, Vol. 3
Spontaneous HTLV-1 transcription is accompanied by distinct epigenetic changes in the 5′ and 3′ long terminal repeats
journal, January 2018
- Miura, Michi; Miyazato, Paola; Satou, Yorifumi
- Wellcome Open Research, Vol. 3
Application of CRISPR/Cas9-Based Gene Editing in HIV-1/AIDS Therapy
journal, March 2019
- Xiao, Qiaoqiao; Guo, Deyin; Chen, Shuliang
- Frontiers in Cellular and Infection Microbiology, Vol. 9
Efficient CRISPR/Cas9 Disruption of Autoimmune-Associated Genes Reveals Key Signaling Programs in Primary Human T Cells
journal, November 2019
- Anderson, Warren; Thorpe, Jerill; Long, S. Alice
- The Journal of Immunology, Vol. 203, Issue 12
Highly Efficient and Versatile Plasmid-Based Gene Editing in Primary T Cells
journal, February 2018
- Kornete, Mara; Marone, Romina; Jeker, Lukas T.
- The Journal of Immunology, Vol. 200, Issue 7
Lessons learned from the blockade of immune checkpoints in cancer immunotherapy
journal, February 2018
- Li, Xiaolei; Shao, Changshun; Shi, Yufang
- Journal of Hematology & Oncology, Vol. 11, Issue 1
Minimal 2'-O-methyl phosphorothioate linkage modification pattern of synthetic guide RNAs for increased stability and efficient CRISPR-Cas9 gene editing avoiding cellular toxicity
journal, November 2017
- Basila, Megan; Kelley, Melissa L.; Smith, Anja van Brabant
- PLOS ONE, Vol. 12, Issue 11
Genetic editing and interrogation with Cpf1 and caged truncated pre-tRNA-like crRNA in mammalian cells
journal, July 2018
- Zhang, Xuhua; Xu, Linping; Fan, Ruihua
- Cell Discovery, Vol. 4, Issue 1
Purity of transferred CD8 + T cells is crucial for safety and efficacy of combinatorial tumor immunotherapy in the absence of SHP‐1
journal, July 2016
- Watson, H. Angharad; Dolton, Garry; Ohme, Julia
- Immunology & Cell Biology, Vol. 94, Issue 8
Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo
journal, August 2018
- Park, Hyo Min; Liu, Hui; Wu, Joann
- Nature Communications, Vol. 9, Issue 1
Allele-specific expression changes dynamically during T cell activation in HLA and other autoimmune loci
journal, February 2020
- Gutierrez-Arcelus, Maria; Baglaenko, Yuriy; Arora, Jatin
- Nature Genetics, Vol. 52, Issue 3
Electroporation of mice zygotes with dual guide RNA/Cas9 complexes for simple and efficient cloning-free genome editing
journal, January 2018
- Teixeira, Marie; Py, Bénédicte F.; Bosc, Christophe
- Scientific Reports, Vol. 8, Issue 1
An Efficient Electroporation Protocol for the Genetic Modification of Mammalian Cells
journal, January 2017
- Chicaybam, Leonardo; Barcelos, Camila; Peixoto, Barbara
- Frontiers in Bioengineering and Biotechnology, Vol. 4
Ribonucleoprotein Transfection for CRISPR/Cas9-Mediated Gene Knockout in Primary T Cells
journal, October 2018
- Oh, Soyoung A.; Seki, Akiko; Rutz, Sascha
- Current Protocols in Immunology, Vol. 124, Issue 1
CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells
journal, April 2017
- Rupp, Levi J.; Schumann, Kathrin; Roybal, Kole T.
- Scientific Reports, Vol. 7, Issue 1
Efficient gene knockin in axolotl and its use to test the role of satellite cells in limb regeneration
journal, October 2017
- Fei, Ji-Feng; Schuez, Maritta; Knapp, Dunja
- Proceedings of the National Academy of Sciences, Vol. 114, Issue 47
CRISPR/Cas9 system targeting regulatory genes of HIV-1 inhibits viral replication in infected T-cell cultures
journal, May 2018
- Ophinni, Youdiil; Inoue, Mari; Kotaki, Tomohiro
- Scientific Reports, Vol. 8, Issue 1
Therapeutic potential of CRISPR/Cas9 gene editing in engineered T‐cell therapy
journal, June 2019
- Gao, Qianqian; Dong, Xuan; Xu, Qumiao
- Cancer Medicine, Vol. 8, Issue 9
Directed evolution of CRISPR-Cas9 to increase its specificity
journal, August 2018
- Lee, Jungjoon K.; Jeong, Euihwan; Lee, Joonsun
- Nature Communications, Vol. 9, Issue 1
Gene Editing of HIV-1 Co-receptors to Prevent and/or Cure Virus Infection
journal, December 2018
- Allen, Alexander G.; Chung, Cheng-Han; Atkins, Andrew
- Frontiers in Microbiology, Vol. 9
Cornerstones of CRISPR–Cas in drug discovery and therapy
journal, December 2016
- Fellmann, Christof; Gowen, Benjamin G.; Lin, Pei-Chun
- Nature Reviews Drug Discovery, Vol. 16, Issue 2
Efficient and versatile CRISPR engineering of human neurons in culture to model neurological disorders
journal, January 2016
- Shah, Ruth R.; Cholewa-Waclaw, Justyna; Davies, Faith C. J.
- Wellcome Open Research, Vol. 1
CRISPR/Cas9-mediated PD-1 disruption enhances human mesothelin-targeted CAR T cell effector functions
journal, December 2018
- Hu, Wanghong; Zi, Zhenguo; Jin, Yanling
- Cancer Immunology, Immunotherapy, Vol. 68, Issue 3
Highly efficient genome editing for single-base substitutions using optimized ssODNs with Cas9-RNPs
journal, March 2019
- Okamoto, Sachiko; Amaishi, Yasunori; Maki, Izumi
- Scientific Reports, Vol. 9, Issue 1
Reprogramming human T cell function and specificity with non-viral genome targeting
journal, July 2018
- Roth, Theodore L.; Puig-Saus, Cristina; Yu, Ruby
- Nature, Vol. 559, Issue 7714
Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci
journal, December 2017
- Lessard, Samuel; Francioli, Laurent; Alfoldi, Jessica
- Proceedings of the National Academy of Sciences, Vol. 114, Issue 52
Light-activated cell identification and sorting (LACIS) for selection of edited clones on a nanofluidic device
journal, May 2018
- Mocciaro, Annamaria; Roth, Theodore L.; Bennett, Hayley M.
- Communications Biology, Vol. 1, Issue 1
Transmembrane E3 ligase RNF183 mediates ER stress-induced apoptosis by degrading Bcl-xL
journal, March 2018
- Wu, Yanfang; Li, Xia; Jia, Junying
- Proceedings of the National Academy of Sciences, Vol. 115, Issue 12
Unexpected heterogeneity derived from Cas9 ribonucleoprotein-introduced clonal cells at the HPRT1 locus
journal, February 2018
- Sakuma, Tetsushi; Mochida, Keiji; Nakade, Shota
- Genes to Cells, Vol. 23, Issue 4
Dynamics of T cell activation mediated by eIF3 interactions with T cell receptor mRNAs
posted_content, October 2020
- De Silva, Dasmanthie; Ferguson, Lucas; Smith, Benjamin E.
- BioRxiv
Cas9 Ribonucleoprotein Complex Delivery: Methods and Applications for Neuroinflammation
journal, June 2019
- Campbell, Lee A.; Richie, Christopher T.; Maggirwar, Nishad S.
- Journal of Neuroimmune Pharmacology, Vol. 14, Issue 4
mRNA as novel technology for passive immunotherapy
journal, October 2018
- Schlake, Thomas; Thess, Andreas; Thran, Moritz
- Cellular and Molecular Life Sciences, Vol. 76, Issue 2
Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair
journal, October 2017
- Lee, Kunwoo; Conboy, Michael; Park, Hyo Min
- Nature Biomedical Engineering, Vol. 1, Issue 11
A genome-wide CRISPR screen identifies a restricted set of HIV host dependency factors
journal, December 2016
- Park, Ryan J.; Wang, Tim; Koundakjian, Dylan
- Nature Genetics, Vol. 49, Issue 2
Methods for Enhancing Clustered Regularly Interspaced Short Palindromic Repeats/Cas9-Mediated Homology-Directed Repair Efficiency
journal, June 2019
- Tang, Xi-Dian; Gao, Fei; Liu, Ming-Jie
- Frontiers in Genetics, Vol. 10
Efficient genome editing of human natural killer cells by CRISPR RNP
posted_content, September 2018
- Rautela, Jai; Surgenor, Elliot; Huntington, Nicholas D.
- BioRxiv
The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy
journal, August 2019
- Sanches-da-Silva, Gabriela De Nardi; Medeiros, Luiza Fonseca Sales; Lima, Fabio Mitsuo
- International Journal of Genomics, Vol. 2019
In vitro transcribed guide RNAs trigger an innate immune response via the RIG-I pathway
journal, March 2018
- Wienert, Beeke; Shin, Jiyung; Zelin, Elena
- PLOS Biology
Editing the Central Nervous System Through CRISPR/Cas9 Systems
journal, May 2019
- Cota-Coronado, Agustin; Díaz-Martínez, Néstor Fabián; Padilla-Camberos, Eduardo
- Frontiers in Molecular Neuroscience, Vol. 12
Regulatory T cells use arginase 2 to enhance their metabolic fitness in tissues
journal, December 2019
- Lowe, Margaret M.; Boothby, Ian; Clancy, Sean
- JCI Insight, Vol. 4, Issue 24
Glucocorticoids paradoxically facilitate steroid resistance in T cell acute lymphoblastic leukemias and thymocytes
journal, December 2019
- Meyer, Lauren K.; Huang, Benjamin J.; Delgado-Martin, Cristina
- Journal of Clinical Investigation, Vol. 130, Issue 2
A novel method to generate T-cell receptor–deficient chimeric antigen receptor T cells
journal, March 2018
- Kamiya, Takahiro; Wong, Desmond; Png, Yi Tian
- Blood Advances, Vol. 2, Issue 5
Guide Swap enables genome-scale pooled CRISPR–Cas9 screening in human primary cells
journal, October 2018
- Ting, Pamela Y.; Parker, Albert E.; Lee, J. Scott
- Nature Methods, Vol. 15, Issue 11
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
journal, August 2018
- Vakulskas, Christopher A.; Dever, Daniel P.; Rettig, Garrett R.
- Nature Medicine, Vol. 24, Issue 8
Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers
journal, August 2017
- Moffett, H. F.; Coon, M. E.; Radtke, S.
- Nature Communications, Vol. 8, Issue 1
The protean world of non-coding RNAs in glioblastoma
journal, May 2019
- Paulmurugan, Ramasamy; Malhotra, Meenakshi; Massoud, Tarik F.
- Journal of Molecular Medicine, Vol. 97, Issue 7
Optimized RNP transfection for highly efficient CRISPR/Cas9-mediated gene knockout in primary T cells
journal, February 2018
- Seki, Akiko; Rutz, Sascha
- Journal of Experimental Medicine, Vol. 215, Issue 3
Dysregulated RASGRP1 expression through RUNX1 mediated transcription promotes autoimmunity
journal, November 2019
- Baars, Matthijs; Douma, Thera; Simeonov, Dimitre R.
- European Journal of Immunology
CRISPR-Cas9 technology: applications and human disease modelling
journal, June 2016
- Torres-Ruiz, Raul; Rodriguez-Perales, Sandra
- Briefings in Functional Genomics, Vol. 16, Issue 1
CRISPR/Cas9-based genome editing in the era of CAR T cell immunotherapy
journal, March 2019
- Salas-Mckee, January; Kong, Weimin; Gladney, Whitney L.
- Human Vaccines & Immunotherapeutics, Vol. 15, Issue 5
In vitro and ex vivo strategies for intracellular delivery
journal, October 2016
- Stewart, Martin P.; Sharei, Armon; Ding, Xiaoyun
- Nature, Vol. 538, Issue 7624
Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration
journal, February 2017
- Kim, Kyoungmi; Park, Sung Wook; Kim, Jin Hyoung
- Genome Research, Vol. 27, Issue 3
CRISPR-Cas9: A cornerstone for the evolution of precision medicine
journal, July 2018
- Razzouk, Sleiman
- Annals of Human Genetics, Vol. 82, Issue 6
CRISPR/Cas9 system: A promising technology for the treatment of inherited and neoplastic hematological diseases
journal, May 2018
- Antony, Justin S.; Haque, A. K. M. Ashiqul; Lamsfus-Calle, Andrés
- Advances in Cell and Gene Therapy, Vol. 1, Issue 1
Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery
journal, December 2015
- Bolukbasi, Mehmet Fatih; Gupta, Ankit; Wolfe, Scot A.
- Nature Methods, Vol. 13, Issue 1
Gene editing enables T-cell engineering to redirect antigen specificity for potent tumor rejection
journal, March 2019
- Albers, Julian J.; Ammon, Tim; Gosmann, Dario
- Life Science Alliance, Vol. 2, Issue 2
Allele-specific expression changes dynamically during T cell activation in HLA and other autoimmune loci
journal, April 2019
- Gutierrez-Arcelus, Maria; Baglaenko, Yuriy; Arora, Jatin
- Nature Genetics
Next Generation CAR T Cells for the Immunotherapy of High-Grade Glioma
journal, February 2019
- Petersen, Christopher T.; Krenciute, Giedre
- Frontiers in Oncology, Vol. 9
The epigenetic landscape of T cell exhaustion
journal, October 2016
- Sen, D. R.; Kaminski, J.; Barnitz, R. A.
- Science, Vol. 354, Issue 6316
Humanized model mice by genome editing and engraftment technologies
journal, June 2018
- Kim, Yong-Sam; Ko, Jeong-Heon
- Molecular & Cellular Toxicology, Vol. 14, Issue 3
Is autoimmunity the Achilles' heel of cancer immunotherapy?
journal, May 2017
- June, Carl H.; Warshauer, Jeremy T.; Bluestone, Jeffrey A.
- Nature Medicine, Vol. 23, Issue 5
T-Cell Exhaustion in Chronic Infections: Reversing the State of Exhaustion and Reinvigorating Optimal Protective Immune Responses
journal, November 2018
- Saeidi, Alireza; Zandi, Keivan; Cheok, Yi Ying
- Frontiers in Immunology, Vol. 9
Resolving mechanisms of immune‐mediated disease in primary CD 4 T cells
journal, April 2020
- Bourges, Christophe; Groff, Abigail F.; Burren, Oliver S.
- EMBO Molecular Medicine, Vol. 12, Issue 5
One-step generation of modular CAR-T cells with AAV–Cpf1
journal, February 2019
- Dai, Xiaoyun; Park, Jonathan J.; Du, Yaying
- Nature Methods, Vol. 16, Issue 3
Generation of tumor antigen-specific murine CD8+ T cells with enhanced anti-tumor activity via highly efficient CRISPR/Cas9 genome editing
journal, February 2018
- Ouchi, Yasuo; Patil, Ashwini; Tamura, Yusuke
- International Immunology, Vol. 30, Issue 4
Chimeric antigen receptor T cell therapy for multiple myeloma
journal, June 2019
- Hasegawa, Kana; Hosen, Naoki
- Inflammation and Regeneration, Vol. 39, Issue 1
The implication of CRISPR/Cas9 genome editing technology in combating human oncoviruses
journal, September 2018
- Gilani, Usman; Shaukat, Memoona; Rasheed, Arisha
- Journal of Medical Virology, Vol. 91, Issue 1
CRISPR–Cas: a tool for cancer research and therapeutics
journal, January 2019
- Yin, Hao; Xue, Wen; Anderson, Daniel G.
- Nature Reviews Clinical Oncology, Vol. 16, Issue 5
Next-generation regulatory T cell therapy
journal, September 2019
- Ferreira, Leonardo M. R.; Muller, Yannick D.; Bluestone, Jeffrey A.
- Nature Reviews Drug Discovery, Vol. 18, Issue 10
Applications of CRISPR technologies in research and beyond
journal, September 2016
- Barrangou, Rodolphe; Doudna, Jennifer A.
- Nature Biotechnology, Vol. 34, Issue 9
An efficient and scalable pipeline for epitope tagging in mammalian stem cells using Cas9 ribonucleoprotein
journal, April 2018
- Dewari, Pooran Singh; Southgate, Benjamin; Mccarten, Katrina
- eLife, Vol. 7
Lipofection-mediated genome editing using DNA-free delivery of the Cas9/gRNA ribonucleoprotein into plant cells
journal, November 2019
- Liu, Wusheng; Rudis, Mary R.; Cheplick, Matthew H.
- Plant Cell Reports, Vol. 39, Issue 2
EfficientCRISPR ‐based genome editing using tandem guideRNA s and editable surrogate reporters
journal, June 2018
- Liu, Wuqing; Li, Shifeng; Zhang, Yunbin
- FEBS Open Bio, Vol. 8, Issue 7
Therapeutic potential of CRISPR/Cas9 gene editing in engineered T‐cell therapy
journal, June 2019
- Gao, Qianqian; Dong, Xuan; Xu, Qumiao
- Cancer Medicine, Vol. 8, Issue 9
Application of Genome Editing Techniques in Immunology
journal, January 2018
- Zych, Agata O.; Bajor, Malgorzata; Zagozdzon, Radoslaw
- Archivum Immunologiae et Therapiae Experimentalis, Vol. 66, Issue 4
Advancing chimeric antigen receptor T cell therapy with CRISPR/Cas9
journal, April 2017
- Ren, Jiangtao; Zhao, Yangbing
- Protein & Cell, Vol. 8, Issue 9
In vitro transcribed sgRNA causes cell death by inducing interferon release
journal, January 2019
- Mu, Wei; Tang, Na; Cheng, Chen
- Protein & Cell, Vol. 10, Issue 6
Recent challenges and advances in genetically-engineered cell therapy
journal, December 2017
- Yong, Seok-Beom; Chung, Jee Young; Song, Yoonsung
- Journal of Pharmaceutical Investigation, Vol. 48, Issue 2
Stem cell-based therapies for HIV/AIDS
journal, August 2016
- Pernet, Olivier; Yadav, Swati Seth; An, Dong Sung
- Advanced Drug Delivery Reviews, Vol. 103
A Cas9 Ribonucleoprotein Platform for Functional Genetic Studies of HIV-Host Interactions in Primary Human T Cells
journal, October 2016
- Hultquist, Judd F.; Schumann, Kathrin; Woo, Jonathan M.
- Cell Reports, Vol. 17, Issue 5
CRISPR/Cas9: a powerful tool for identification of new targets for cancer treatment
journal, April 2019
- Liu, Bin; Saber, Ali; Haisma, Hidde J.
- Drug Discovery Today, Vol. 24, Issue 4
CRISPR-mediated genome editing and human diseases
journal, December 2016
- Cai, Liquan; Fisher, Alfred L.; Huang, Haochu
- Genes & Diseases, Vol. 3, Issue 4
CRISPR-Mediated Editing of the B Cell Receptor in Primary Human B Cells
journal, February 2019
- Greiner, Vera; Bou Puerto, Regina; Liu, Suying
- iScience, Vol. 12
Versatility of chemically synthesized guide RNAs for CRISPR-Cas9 genome editing
journal, September 2016
- Kelley, Melissa L.; Strezoska, Žaklina; He, Kaizhang
- Journal of Biotechnology, Vol. 233
Delivery of Cas9 Protein into Mouse Zygotes through a Series of Electroporation Dramatically Increases the Efficiency of Model Creation
journal, May 2016
- Wang, Wenbo; Kutny, Peter M.; Byers, Shannon L.
- Journal of Genetics and Genomics, Vol. 43, Issue 5
CRISPR/Cas9: Transcending the Reality of Genome Editing
journal, June 2017
- Chira, Sergiu; Gulei, Diana; Hajitou, Amin
- Molecular Therapy - Nucleic Acids, Vol. 7
CRISPR/Cas9-Directed Reassignment of the GATA1 Initiation Codon in K562 Cells to Recapitulate AML in Down Syndrome
journal, June 2017
- Bloh, Kevin M.; Bialk, Pawel A.; Gopalakrishnapillai, Anilkumar
- Molecular Therapy - Nucleic Acids, Vol. 7
VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9
journal, September 2018
- Montagna, Claudia; Petris, Gianluca; Casini, Antonio
- Molecular Therapy - Nucleic Acids, Vol. 12
Transient Retrovirus-Based CRISPR/Cas9 All-in-One Particles for Efficient, Targeted Gene Knockout
journal, December 2018
- Knopp, Yvonne; Geis, Franziska K.; Heckl, Dirk
- Molecular Therapy - Nucleic Acids, Vol. 13
Efficient Gene Editing at Major CFTR Mutation Loci
journal, June 2019
- Ruan, Jinxue; Hirai, Hiroyuki; Yang, Dongshan
- Molecular Therapy - Nucleic Acids, Vol. 16
Simplified CRISPR tools for efficient genome editing and streamlined protocols for their delivery into mammalian cells and mouse zygotes
journal, May 2017
- Jacobi, Ashley M.; Rettig, Garrett R.; Turk, Rolf
- Methods, Vol. 121-122
Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells
journal, April 2017
- MacLeod, Daniel T.; Antony, Jeyaraj; Martin, Aaron J.
- Molecular Therapy, Vol. 25, Issue 4
Genome-editing Technologies for Gene and Cell Therapy
journal, March 2016
- Maeder, Morgan L.; Gersbach, Charles A.
- Molecular Therapy, Vol. 24, Issue 3
Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers
journal, August 2017
- Moffett, H. F.; Coon, M. E.; Radtke, S.
- Nature Communications, Vol. 8, Issue 1
Extension of the crRNA enhances Cpf1 gene editing in vitro and in vivo
journal, August 2018
- Park, Hyo Min; Liu, Hui; Wu, Joann
- Nature Communications, Vol. 9, Issue 1
Reprogramming human T cell function and specificity with non-viral genome targeting
journal, July 2018
- Roth, Theodore L.; Puig-Saus, Cristina; Yu, Ruby
- Nature, Vol. 559, Issue 7714
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells
journal, August 2018
- Vakulskas, Christopher A.; Dever, Daniel P.; Rettig, Garrett R.
- Nature Medicine, Vol. 24, Issue 8
CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells
journal, April 2017
- Rupp, Levi J.; Schumann, Kathrin; Roybal, Kole T.
- Scientific Reports, Vol. 7, Issue 1
Using nanoBRET and CRISPR/Cas9 to monitor proximity to a genome-edited protein in real-time
journal, June 2017
- White, Carl W.; Vanyai, Hannah K.; See, Heng B.
- Scientific Reports, Vol. 7, Issue 1
CRISPR/Cas9 editing reveals novel mechanisms of clustered microRNA regulation and function
journal, August 2017
- Lataniotis, Lazaros; Albrecht, Andreas; Kok, Fatma O.
- Scientific Reports, Vol. 7, Issue 1
Simultaneous electroporation and dielectrophoresis in non-electrolytic micro/nano-electroporation
journal, February 2018
- Lyu, Chenang; Wang, Jianping; Powell-Palm, Matthew
- Scientific Reports, Vol. 8, Issue 1
CRISPR/Cas9 system targeting regulatory genes of HIV-1 inhibits viral replication in infected T-cell cultures
journal, May 2018
- Ophinni, Youdiil; Inoue, Mari; Kotaki, Tomohiro
- Scientific Reports, Vol. 8, Issue 1
Efficient homology-directed gene editing by CRISPR/Cas9 in human stem and primary cells using tube electroporation
journal, August 2018
- Xu, Xiaoyun; Gao, Dongbing; Wang, Ping
- Scientific Reports, Vol. 8, Issue 1
A comparison of inverted and upright laser-activated titanium nitride micropyramids for intracellular delivery
journal, October 2018
- Raun, Alexander; Saklayen, Nabiha; Zgrabik, Christine
- Scientific Reports, Vol. 8, Issue 1
Co-expression of Cas9 and single-guided RNAs in Escherichia coli streamlines production of Cas9 ribonucleoproteins
journal, May 2019
- Qiao, Jie; Li, Wenqiang; Lin, Siyu
- Communications Biology, Vol. 2, Issue 1
Understanding the diversity of genetic outcomes from CRISPR-Cas generated homology-directed repair
journal, December 2019
- Sansbury, Brett M.; Hewes, Amanda M.; Kmiec, Eric B.
- Communications Biology, Vol. 2, Issue 1
Analyses of point mutation repair and allelic heterogeneity generated by CRISPR/Cas9 and single-stranded DNA oligonucleotides
journal, September 2016
- Bialk, Pawel; Sansbury, Brett; Rivera-Torres, Natalia
- Scientific Reports, Vol. 6, Issue 1
A vector platform for the rapid and efficient engineering of stable complex transgenes
journal, October 2016
- Jäckel, Carsten; Nogueira, Melanie Schmitt; Ehni, Nadja
- Scientific Reports, Vol. 6, Issue 1
GapmeR cellular internalization by macropinocytosis induces sequence-specific gene silencing in human primary T-cells
journal, November 2016
- Fazil, Mobashar Hussain Urf Turabe; Ong, Seow Theng; Chalasani, Madhavi Latha Somaraju
- Scientific Reports, Vol. 6, Issue 1
Reprogramming MHC specificity by CRISPR-Cas9-assisted cassette exchange
journal, April 2017
- Kelton, William; Waindok, Ann Cathrin; Pesch, Theresa
- Scientific Reports, Vol. 7, Issue 1
Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci
journal, December 2017
- Lessard, Samuel; Francioli, Laurent; Alfoldi, Jessica
- Proceedings of the National Academy of Sciences, Vol. 114, Issue 52
Transmembrane E3 ligase RNF183 mediates ER stress-induced apoptosis by degrading Bcl-xL
journal, March 2018
- Wu, Yanfang; Li, Xia; Jia, Junying
- Proceedings of the National Academy of Sciences, Vol. 115, Issue 12
Metabolic reprogramming of human CD8 + memory T cells through loss of SIRT1
journal, November 2017
- Jeng, Mark Y.; Hull, Philip A.; Fei, Mingjian
- The Journal of Experimental Medicine, Vol. 215, Issue 1
Optimized RNP transfection for highly efficient CRISPR/Cas9-mediated gene knockout in primary T cells
journal, February 2018
- Seki, Akiko; Rutz, Sascha
- Journal of Experimental Medicine, Vol. 215, Issue 3
CRISPRs for Optimal Targeting: Delivery of CRISPR Components as DNA, RNA, and Protein into Cultured Cells and Single-Cell Embryos
journal, June 2016
- Kouranova, Evguenia; Forbes, Kevin; Zhao, Guojun
- Human Gene Therapy, Vol. 27, Issue 6
Discovery of an autoimmunity-associated IL2RA enhancer by unbiased targeting of transcriptional activation
posted_content, December 2016
- Simeonov, Dimitre R.; Gowen, Benjamin G.; Boontanrart, Mandy
- BioRxiv
Rewired signaling network in T cells expressing the chimeric antigen receptor (CAR)
journal, December 2019
- Dong, Rui; Libby, Kendra A.; Blaeschke, Franziska
- The EMBO Journal
An efficient and scalable pipeline for epitope tagging in mammalian stem cells using Cas9 ribonucleoprotein
journal, January 2018
- Dewari, Pooran Singh; Southgate, Benjamin; Mccarten, Katrina
- eLife
The human leukemia virus HTLV-1 alters the structure and transcription of host chromatin in cis
journal, March 2018
- Melamed, Anat; Yaguchi, Hiroko; Miura, Michi
- eLife
Mapping gene regulatory networks of primary CD4+ T cells using single-cell genomics and genome engineering
posted_content, June 2019
- Gate, Rachel E.; Kim, Min Cheol; Lu, Andrew
- BioRxiv
Carboxylated branched poly(β-amino ester) nanoparticles enable robust cytosolic protein delivery and CRISPR-Cas9 gene editing
journal, December 2019
- Rui, Yuan; Wilson, David R.; Choi, John
- Science Advances, Vol. 5, Issue 12
A CRISPR-Based Toolbox for Studying T Cell Signal Transduction
journal, January 2016
- Chi, Shen; Weiss, Arthur; Wang, Haopeng
- BioMed Research International, Vol. 2016
The Potential Use of the CRISPR-Cas System for HIV-1 Gene Therapy
journal, August 2019
- Sanches-da-Silva, Gabriela De Nardi; Medeiros, Luiza Fonseca Sales; Lima, Fabio Mitsuo
- International Journal of Genomics, Vol. 2019
Primary allogeneic mitochondrial mix (PAMM) transfer/transplant by MitoCeption to address damage in PBMCs caused by ultraviolet radiation
journal, June 2019
- Cabrera, Francisco; Ortega, Mayra; Velarde, Francesca
- BMC Biotechnology, Vol. 19, Issue 1
Translational research—the need of a new bioethics approach
journal, January 2016
- Hostiuc, Sorin; Moldoveanu, Alin; Dascălu, Maria-Iuliana
- Journal of Translational Medicine, Vol. 14, Issue 1
Lessons learned from the blockade of immune checkpoints in cancer immunotherapy
journal, February 2018
- Li, Xiaolei; Shao, Changshun; Shi, Yufang
- Journal of Hematology & Oncology, Vol. 11, Issue 1
Efficient precise knockin with a double cut HDR donor after CRISPR/Cas9-mediated double-stranded DNA cleavage
journal, February 2017
- Zhang, Jian-Ping; Li, Xiao-Lan; Li, Guo-Hua
- Genome Biology, Vol. 18, Issue 1
Strategies for precision modulation of gene expression by epigenome editing: an overview
journal, September 2015
- Laufer, Benjamin I.; Singh, Shiva M.
- Epigenetics & Chromatin, Vol. 8, Issue 1
Chimeric antigen receptor T cell therapy for multiple myeloma
journal, June 2019
- Hasegawa, Kana; Hosen, Naoki
- Inflammation and Regeneration, Vol. 39, Issue 1
Targeting the latent reservoir to achieve functional HIV cure
journal, May 2016
- Cary, Daniele C.; Peterlin, B. Matija
- F1000Research, Vol. 5
Epigenetic changes around the pX region and spontaneous HTLV-1 transcription are CTCF-independent
journal, January 2018
- Miura, Michi; Miyazato, Paola; Satou, Yorifumi
- Wellcome Open Research, Vol. 3
In vitro–transcribed guide RNAs trigger an innate immune response via the RIG-I pathway
journal, July 2018
- Wienert, Beeke; Shin, Jiyung; Zelin, Elena
- PLOS Biology, Vol. 16, Issue 7
CRISPR/Cas-mediated knock-in via non-homologous end-joining in the crustacean Daphnia magna
journal, October 2017
- Kumagai, Hitoshi; Nakanishi, Takashi; Matsuura, Tomoaki
- PLOS ONE, Vol. 12, Issue 10
A versatile system for rapid multiplex genome-edited CAR T cell generation
journal, February 2017
- Ren, Jiangtao; Zhang, Xuhua; Liu, Xiaojun
- Oncotarget, Vol. 8, Issue 10
Gene editing enables T-cell engineering to redirect antigen specificity for potent tumor rejection
journal, March 2019
- Albers, Julian J.; Ammon, Tim; Gosmann, Dario
- Life Science Alliance, Vol. 2, Issue 2
Application of CRISPR/Cas9-Based Gene Editing in HIV-1/AIDS Therapy
journal, March 2019
- Xiao, Qiaoqiao; Guo, Deyin; Chen, Shuliang
- Frontiers in Cellular and Infection Microbiology, Vol. 9
Gene Editing of HIV-1 Co-receptors to Prevent and/or Cure Virus Infection
journal, December 2018
- Allen, Alexander G.; Chung, Cheng-Han; Atkins, Andrew
- Frontiers in Microbiology, Vol. 9
Editing the Central Nervous System Through CRISPR/Cas9 Systems
journal, May 2019
- Cota-Coronado, Agustin; Díaz-Martínez, Néstor Fabián; Padilla-Camberos, Eduardo
- Frontiers in Molecular Neuroscience, Vol. 12
CRISPR-Cas9: A Revolutionary Tool for Cancer Modelling
journal, September 2015
- Torres-Ruiz, Raul; Rodriguez-Perales, Sandra
- International Journal of Molecular Sciences, Vol. 16, Issue 9
RNA Interference in the Age of CRISPR: Will CRISPR Interfere with RNAi?
journal, February 2016
- Unniyampurath, Unnikrishnan; Pilankatta, Rajendra; Krishnan, Manoj
- International Journal of Molecular Sciences, Vol. 17, Issue 3
Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
journal, May 2018
- Farboud, Behnom; Jarvis, Erin; Roth, Theodore L.
- Journal of Visualized Experiments, Issue 135
CRISPR therapeutic tools for complex genetic disorders and cancer (Review)
journal, June 2018
- Baliou, Stella; Adamaki, Maria; Kyriakopoulos, Anthony
- International Journal of Oncology
An Overview Of The Crispr-Based Genomic- And Epigenome-Editing System: Function, Applications, And Challenges
journal, January 2019
- Mojarrad, Majid; Bozorg Qomi, Saeed; Asghari, Amir
- Advanced Biomedical Research, Vol. 8, Issue 1
Efficient And Versatile Crispr Engineering Of Human Neurons In Culture To Model Neurological Disorders
text, January 2016
- Shah, Ruth Rama; Cholewa-Waclaw, Justyna; Davies, Faith C. J.
- Zenodo
Correction: Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
journal, November 2018
- Bak, Rasmus O.; Dever, Daniel P.; Reinisch, Andreas
- eLife, Vol. 7