gene editing

Summary

Summary: Genetic engineering techniques that involve DNA REPAIR mechanisms for incorporating site-specific modifications into a cell's genome.

Top Publications

  1. Richter M, Stone D, MIAO C, Humbert O, Kiem H, Papayannopoulou T, et al. In Vivo Hematopoietic Stem Cell Transduction. Hematol Oncol Clin North Am. 2017;31:771-785 pubmed publisher
    ..We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages. ..
  2. Ormond K, Mortlock D, Scholes D, Bombard Y, Brody L, Faucett W, et al. Human Germline Genome Editing. Am J Hum Genet. 2017;101:167-176 pubmed publisher
    ..and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy...
  3. Zhao W, Siegel D, Biton A, Tonqueze O, Zaitlen N, Ahituv N, et al. CRISPR-Cas9-mediated functional dissection of 3'-UTRs. Nucleic Acids Res. 2017;45:10800-10810 pubmed publisher
    ..Due to intrinsic limitations of heterologous reporter assays, we sought to develop a gene editing approach to investigate the regulatory activity of 3'-UTRs in their native context...
  4. Hess G, Tycko J, Yao D, Bassik M. Methods and Applications of CRISPR-Mediated Base Editing in Eukaryotic Genomes. Mol Cell. 2017;68:26-43 pubmed publisher
    ..Additionally, we discuss how these systems have been applied in therapeutic, engineering, and research settings. Lastly, we explore future directions of this emerging technology. ..
  5. Adair J, Kubek S, Kiem H. Hematopoietic Stem Cell Approaches to Cancer. Hematol Oncol Clin North Am. 2017;31:897-912 pubmed publisher
    ..We also discuss how HSCs can be harnessed to produce powerful tumor killing T cells, potentially benefitting and complementing T-cell-based immunotherapies. ..
  6. Engelholm L, Riaz A, Serra D, Dagnæs Hansen F, Johansen J, Santoni Rugiu E, et al. CRISPR/Cas9 Engineering of Adult Mouse Liver Demonstrates That the Dnajb1-Prkaca Gene Fusion Is Sufficient to Induce Tumors Resembling Fibrolamellar Hepatocellular Carcinoma. Gastroenterology. 2017;153:1662-1673.e10 pubmed publisher
    ..Strategies to block DNAJB1-PRKACA might be developed as therapeutics for this form of liver cancer. ..
  7. Liang P, Sun H, Sun Y, Zhang X, Xie X, Zhang J, et al. Effective gene editing by high-fidelity base editor 2 in mouse zygotes. Protein Cell. 2017;8:601-611 pubmed publisher
    ..Taken together, our work demonstrates the feasibility of generating point mutations in mouse by base editing, and underscores the need to carefully optimize base editing systems in order to eliminate proximal-site deamination. ..
  8. Shimatani Z, Kashojiya S, Takayama M, Terada R, Arazoe T, Ishii H, et al. Targeted base editing in rice and tomato using a CRISPR-Cas9 cytidine deaminase fusion. Nat Biotechnol. 2017;35:441-443 pubmed publisher
  9. Gupta R, Hadaya J, Trehan A, Zekavat S, Roselli C, Klarin D, et al. A Genetic Variant Associated with Five Vascular Diseases Is a Distal Regulator of Endothelin-1 Gene Expression. Cell. 2017;170:522-533.e15 pubmed publisher
  10. Rose J, Stephany J, Valente W, Trevillian B, Dang H, Bielas J, et al. Rapidly inducible Cas9 and DSB-ddPCR to probe editing kinetics. Nat Methods. 2017;14:891-896 pubmed publisher
    ..However, we observe distinct kinetic profiles, even for proximal sites, and this suggests that target sequence and chromatin state modulate cleavage and repair kinetics. ..

Detail Information

Publications98

  1. Richter M, Stone D, MIAO C, Humbert O, Kiem H, Papayannopoulou T, et al. In Vivo Hematopoietic Stem Cell Transduction. Hematol Oncol Clin North Am. 2017;31:771-785 pubmed publisher
    ..We provide examples for in vivo HSC gene therapy and discuss advantages and disadvantages. ..
  2. Ormond K, Mortlock D, Scholes D, Bombard Y, Brody L, Faucett W, et al. Human Germline Genome Editing. Am J Hum Genet. 2017;101:167-176 pubmed publisher
    ..and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy...
  3. Zhao W, Siegel D, Biton A, Tonqueze O, Zaitlen N, Ahituv N, et al. CRISPR-Cas9-mediated functional dissection of 3'-UTRs. Nucleic Acids Res. 2017;45:10800-10810 pubmed publisher
    ..Due to intrinsic limitations of heterologous reporter assays, we sought to develop a gene editing approach to investigate the regulatory activity of 3'-UTRs in their native context...
  4. Hess G, Tycko J, Yao D, Bassik M. Methods and Applications of CRISPR-Mediated Base Editing in Eukaryotic Genomes. Mol Cell. 2017;68:26-43 pubmed publisher
    ..Additionally, we discuss how these systems have been applied in therapeutic, engineering, and research settings. Lastly, we explore future directions of this emerging technology. ..
  5. Adair J, Kubek S, Kiem H. Hematopoietic Stem Cell Approaches to Cancer. Hematol Oncol Clin North Am. 2017;31:897-912 pubmed publisher
    ..We also discuss how HSCs can be harnessed to produce powerful tumor killing T cells, potentially benefitting and complementing T-cell-based immunotherapies. ..
  6. Engelholm L, Riaz A, Serra D, Dagnæs Hansen F, Johansen J, Santoni Rugiu E, et al. CRISPR/Cas9 Engineering of Adult Mouse Liver Demonstrates That the Dnajb1-Prkaca Gene Fusion Is Sufficient to Induce Tumors Resembling Fibrolamellar Hepatocellular Carcinoma. Gastroenterology. 2017;153:1662-1673.e10 pubmed publisher
    ..Strategies to block DNAJB1-PRKACA might be developed as therapeutics for this form of liver cancer. ..
  7. Liang P, Sun H, Sun Y, Zhang X, Xie X, Zhang J, et al. Effective gene editing by high-fidelity base editor 2 in mouse zygotes. Protein Cell. 2017;8:601-611 pubmed publisher
    ..Taken together, our work demonstrates the feasibility of generating point mutations in mouse by base editing, and underscores the need to carefully optimize base editing systems in order to eliminate proximal-site deamination. ..
  8. Shimatani Z, Kashojiya S, Takayama M, Terada R, Arazoe T, Ishii H, et al. Targeted base editing in rice and tomato using a CRISPR-Cas9 cytidine deaminase fusion. Nat Biotechnol. 2017;35:441-443 pubmed publisher
  9. Gupta R, Hadaya J, Trehan A, Zekavat S, Roselli C, Klarin D, et al. A Genetic Variant Associated with Five Vascular Diseases Is a Distal Regulator of Endothelin-1 Gene Expression. Cell. 2017;170:522-533.e15 pubmed publisher
  10. Rose J, Stephany J, Valente W, Trevillian B, Dang H, Bielas J, et al. Rapidly inducible Cas9 and DSB-ddPCR to probe editing kinetics. Nat Methods. 2017;14:891-896 pubmed publisher
    ..However, we observe distinct kinetic profiles, even for proximal sites, and this suggests that target sequence and chromatin state modulate cleavage and repair kinetics. ..
  11. Kim D, Lim K, Kim S, Yoon S, Kim K, Ryu S, et al. Genome-wide target specificities of CRISPR RNA-guided programmable deaminases. Nat Biotechnol. 2017;35:475-480 pubmed publisher
    ..1%. Notably, off-target sites of the base editors are often different from those of Cas9 alone, calling for independent assessment of their genome-wide specificities. ..
  12. Lux C, Scharenberg A. Therapeutic Gene Editing Safety and Specificity. Hematol Oncol Clin North Am. 2017;31:787-795 pubmed publisher
    Therapeutic gene editing is significant for medical advancement. Safety is intricately linked to the specificity of the editing tools used to cut at precise genomic targets...
  13. Kuan P, Powers S, He S, Li K, Zhao X, Huang B. A systematic evaluation of nucleotide properties for CRISPR sgRNA design. BMC Bioinformatics. 2017;18:297 pubmed publisher
    CRISPR is a versatile gene editing tool which has revolutionized genetic research in the past few years. Optimizing sgRNA design to improve the efficiency of target/DNA cleavage is critical to ensure the success of CRISPR screens...
  14. Liang P, Ding C, Sun H, Xie X, Xu Y, Zhang X, et al. Correction of ?-thalassemia mutant by base editor in human embryos. Protein Cell. 2017;8:811-822 pubmed publisher
    ..Collectively, this study demonstrated the feasibility of curing genetic disease in human somatic cells and embryos by base editor system. ..
  15. Yang S, Chang R, Yang H, Zhao T, Hong Y, Kong H, et al. CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease. J Clin Invest. 2017;127:2719-2724 pubmed publisher
    ..Our studies suggest that non-allele-specific CRISPR/Cas9-mediated gene editing could be used to efficiently and permanently eliminate polyglutamine expansion-mediated neuronal toxicity in ..
  16. Cinghu S, Yang P, Kosak J, Conway A, Kumar D, Oldfield A, et al. Intragenic Enhancers Attenuate Host Gene Expression. Mol Cell. 2017;68:104-117.e6 pubmed publisher
  17. Hindriksen S, Bramer A, Truong M, Vromans M, Post J, Verlaan Klink I, et al. Baculoviral delivery of CRISPR/Cas9 facilitates efficient genome editing in human cells. PLoS ONE. 2017;12:e0179514 pubmed publisher
    ..Tagging of the CPC recruitment factor Haspin with the fluorescent reporter YFP allowed us to study its native localization as well as recruitment to the cohesin subunit Pds5B. ..
  18. Yamazaki T, Hatano Y, Handa T, Kato S, Hoida K, Yamamura R, et al. Targeted DNA methylation in pericentromeres with genome editing-based artificial DNA methyltransferase. PLoS ONE. 2017;12:e0177764 pubmed publisher
    ..We have demonstrated a new method of introducing DNA methylation without the need of other binding partners using the CpG methyltransferase, SssI. ..
  19. Abadi S, Yan W, Amar D, Mayrose I. A machine learning approach for predicting CRISPR-Cas9 cleavage efficiencies and patterns underlying its mechanism of action. PLoS Comput Biol. 2017;13:e1005807 pubmed publisher
    ..We discover that attributes that describe the spatial structure and rigidity of the entire genomic site as well as those surrounding the PAM region are a major component of the prediction capabilities...
  20. Shao S, Ren C, Liu Z, Bai Y, Chen Z, Wei Z, et al. Enhancing CRISPR/Cas9-mediated homology-directed repair in mammalian cells by expressing Saccharomyces cerevisiae Rad52. Int J Biochem Cell Biol. 2017;92:43-52 pubmed publisher
    ..2-fold increase in HDR frequency. In conclusion, our data suggests that Rad52-Cas9 fusion is a good option for enhancing CRISPR/Cas9-mediated HDR, which may be of use in future studies involving precise genome editing. ..
  21. Gandhi S, Haeussler M, Razy Krajka F, Christiaen L, Stolfi A. Evaluation and rational design of guide RNAs for efficient CRISPR/Cas9-mediated mutagenesis in Ciona. Dev Biol. 2017;425:8-20 pubmed publisher
    ..We anticipate that our approach can be scaled up to systematically design and deliver highly efficient sgRNAs for the tissue-specific investigation of gene functions in Ciona. ..
  22. Aslan Y, Tadjuidje E, Zorn A, Cha S. High-efficiency non-mosaic CRISPR-mediated knock-in and indel mutation in F0 Xenopus. Development. 2017;144:2852-2858 pubmed publisher
    ..in animal models still presents a number of challenges, including the time to establish mutant lines, mosaic gene editing in founder animals, and low homologous recombination rates...
  23. Cong L. CRISPR: Groundbreaking technology for RNA-guided genome engineering. Anal Biochem. 2017;532:87-89 pubmed publisher
  24. Agudelo D, Duringer A, Bozoyan L, Huard C, Carter S, Loehr J, et al. Marker-free coselection for CRISPR-driven genome editing in human cells. Nat Methods. 2017;14:615-620 pubmed publisher
    ..The use of universal CRISPR reagents and a commercially available small-molecule inhibitor streamlines the incorporation of marker-free genetic changes in human cells. ..
  25. Weh E, Takeuchi H, Muheisen S, Haltiwanger R, Semina E. Functional characterization of zebrafish orthologs of the human Beta 3-Glucosyltransferase B3GLCT gene mutated in Peters Plus Syndrome. PLoS ONE. 2017;12:e0184903 pubmed publisher
    ..At the same time, complete b3glct deficiency in zebrafish appears to be inconsequential and possibly compensated for by a yet unknown mechanism. ..
  26. Billon P, Bryant E, Joseph S, Nambiar T, Hayward S, Rothstein R, et al. CRISPR-Mediated Base Editing Enables Efficient Disruption of Eukaryotic Genes through Induction of STOP Codons. Mol Cell. 2017;67:1068-1079.e4 pubmed publisher
    ..Additionally, our database includes sgSTOPs that could be employed to precisely model over 32,000 cancer-associated nonsense mutations. Altogether, this work provides a comprehensive resource for DSB-free gene disruption by iSTOP. ..
  27. Zhou W, Wan Y, Guo R, Deng M, Deng K, Wang Z, et al. Generation of beta-lactoglobulin knock-out goats using CRISPR/Cas9. PLoS ONE. 2017;12:e0186056 pubmed publisher
    ..Our study thus provides a basis for optimizing the quality of goat milk, which can be applied to biomedical and agricultural research...
  28. Cohen J. CRISPR patent ruling leaves license holders scrambling. Science. 2017;355:786 pubmed publisher
  29. Gundry M, Dever D, Yudovich D, Bauer D, Haas S, Wilkinson A, et al. Technical considerations for the use of CRISPR/Cas9 in hematology research. Exp Hematol. 2017;54:4-11 pubmed publisher
    ..We also summarize the technical considerations and advice provided during the May 2017 International Society of Experimental Hematology New Investigator Committee webinar on the same topic. ..
  30. Huang J, Li K, Cai W, Liu X, Zhang Y, Orkin S, et al. Dissecting super-enhancer hierarchy based on chromatin interactions. Nat Commun. 2018;9:943 pubmed publisher
    ..Genetic ablation of hub enhancers results in profound defects in gene activation and local chromatin landscape. As such, hub enhancers are the major constituents responsible for SE functional and structural organization. ..
  31. Swarts D, van der Oost J, Jinek M. Structural Basis for Guide RNA Processing and Seed-Dependent DNA Targeting by CRISPR-Cas12a. Mol Cell. 2017;66:221-233.e4 pubmed publisher
    ..Together, these insights advance our mechanistic understanding of Cas12a enzymes and may contribute to further development of genome editing technologies. ..
  32. Lucas D, O Leary H, Ebert B, Cowan C, Tremblay C. Utility of CRISPR/Cas9 systems in hematology research. Exp Hematol. 2017;54:1-3 pubmed publisher
    ..Here, we provide an overview of the topics they covered, including their insights into the novel applications of the technique and its strengths and limitations. ..
  33. Haarhuis J, van der Weide R, Blomen V, Yáñez Cuna J, Amendola M, van Ruiten M, et al. The Cohesin Release Factor WAPL Restricts Chromatin Loop Extension. Cell. 2017;169:693-707.e14 pubmed publisher
    ..Finally, we find that whereas cohesin promotes chromosomal looping, it rather limits nuclear compartmentalization. We conclude that the balanced activity of SCC2/SCC4 and WAPL enables cohesin to correctly structure chromosomes. ..
  34. Khatodia S, Bhatotia K, Tuteja N. Development of CRISPR/Cas9 mediated virus resistance in agriculturally important crops. Bioengineered. 2017;8:274-279 pubmed publisher
    ..In conclusion, CRISPR/Cas9 technology will provide a more durable and broad spectrum viral resistance in agriculturally important crops which will eventually lead to public acceptance and commercialization in the near future. ..
  35. Tu M, Lin L, Cheng Y, He X, Sun H, Xie H, et al. A 'new lease of life': FnCpf1 possesses DNA cleavage activity for genome editing in human cells. Nucleic Acids Res. 2017;45:11295-11304 pubmed publisher
    ..Our study identifies FnCpf1 as a new member of the Cpf1 family for human genome editing with distinctive characteristics, which shows promise as a genome editing tool with the potential for both research and therapeutic applications. ..
  36. Zhang H, Shi J, Hachet M, Xue C, Bauer R, Jiang H, et al. CRISPR/Cas9-Mediated Gene Editing in Human iPSC-Derived Macrophage Reveals Lysosomal Acid Lipase Function in Human Macrophages-Brief Report. Arterioscler Thromb Vasc Biol. 2017;37:2156-2160 pubmed publisher
    ..CRISPR/Cas9 and IPSDM provide important tools to study human macrophage biology and more broadly for future studies of disease-associated LIPA genetic variation in human macrophages. ..
  37. O Geen H, Ren C, Nicolet C, Perez A, Halmai J, Le V, et al. dCas9-based epigenome editing suggests acquisition of histone methylation is not sufficient for target gene repression. Nucleic Acids Res. 2017;45:9901-9916 pubmed publisher
    ..The easily programmable dCas9 toolkit allowed precise control of epigenetic information and dissection of the relationship between the epigenome and gene regulation...
  38. Johansen A, Molenaar B, Versteeg D, Leitoguinho A, Demkes C, Spanjaard B, et al. Postnatal Cardiac Gene Editing Using CRISPR/Cas9 With AAV9-Mediated Delivery of Short Guide RNAs Results in Mosaic Gene Disruption. Circ Res. 2017;121:1168-1181 pubmed publisher
    ..CRISPR/Cas9 has been extensively used to manipulate the germline in zygotes, its application in postnatal gene editing remains incompletely characterized...
  39. Kim Y, Cheong S, Lee J, Lee S, Lee M, Baek I, et al. Generation of knockout mice by Cpf1-mediated gene targeting. Nat Biotechnol. 2016;34:808-10 pubmed publisher
  40. Chadwick A, Wang X, Musunuru K. In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing. Arterioscler Thromb Vasc Biol. 2017;37:1741-1747 pubmed publisher
  41. Wolfs E, Holvoet B, Ordovas L, Breuls N, Helsen N, Schönberger M, et al. Molecular Imaging of Human Embryonic Stem Cells Stably Expressing Human PET Reporter Genes After Zinc Finger Nuclease-Mediated Genome Editing. J Nucl Med. 2017;58:1659-1665 pubmed publisher
    ..b>Conclusion: This study demonstrates the application of genome editing for targeted integration of human imaging reporter genes in human embryonic stem cells for long-term molecular imaging. ..
  42. Murugan K, Babu K, Sundaresan R, Rajan R, Sashital D. The Revolution Continues: Newly Discovered Systems Expand the CRISPR-Cas Toolkit. Mol Cell. 2017;68:15-25 pubmed publisher
    ..These advances highlight the exciting prospects for future tools based on the continually expanding set of CRISPR-Cas systems...
  43. Nishimasu H, Yamano T, Gao L, Zhang F, Ishitani R, Nureki O. Structural Basis for the Altered PAM Recognition by Engineered CRISPR-Cpf1. Mol Cell. 2017;67:139-147.e2 pubmed publisher
    ..Our high-resolution structures delineated the altered PAM recognition mechanisms of the AsCpf1 variants, providing a basis for the further engineering of CRISPR-Cpf1. ..
  44. van Tienen L, Mieszczanek J, Fiedler M, Rutherford T, Bienz M. Constitutive scaffolding of multiple Wnt enhanceosome components by Legless/BCL9. elife. 2017;6: pubmed publisher
    ..An unexpected link between BCL9/B9L, PYGO2 and nuclear co-receptor complexes suggests that these β-catenin co-factors may coordinate Wnt and nuclear hormone responses. ..
  45. Stella S, Alcón P, Montoya G. Structure of the Cpf1 endonuclease R-loop complex after target DNA cleavage. Nature. 2017;546:559-563 pubmed publisher
    ..Mutations in key residues reveal a mechanism linking the PAM and DNA nuclease sites. Analysis of the Cpf1 structures proposes a singular working model of RNA-guided DNA cleavage, suggesting new avenues for redesign of Cpf1. ..
  46. Li H, Liu G. The application of CRISPR/Cas9 in genome editing of filamentous fungi. Yi Chuan. 2017;39:355-367 pubmed publisher
    ..This review summarizes the application of CRISPR/Cas9 in genome editing of filamentous fungi and aims to provide a reference for the research in this field. ..
  47. Swiat M, Dashko S, den Ridder M, Wijsman M, van der Oost J, Daran J, et al. FnCpf1: a novel and efficient genome editing tool for Saccharomyces cerevisiae. Nucleic Acids Res. 2017;45:12585-12598 pubmed publisher
    ..cerevisiae. FnCpf1 proves to be a powerful addition to S. cerevisiae CRISPR toolbox. ..
  48. Kohn D. Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy. Hematol Oncol Clin North Am. 2017;31:721-735 pubmed publisher
    ..b>Gene editing using site-specific endonucleases is emerging as a promising technology for gene therapy and is moving into ..
  49. Nakamae K, Nishimura Y, Takenaga M, Nakade S, Sakamoto N, Ide H, et al. Establishment of expanded and streamlined pipeline of PITCh knock-in - a web-based design tool for MMEJ-mediated gene knock-in, PITCh designer, and the variations of PITCh, PITCh-TG and PITCh-KIKO. Bioengineered. 2017;8:302-308 pubmed publisher
  50. Pankowicz F, Jarrett K, Lagor W, Bissig K. CRISPR/Cas9: at the cutting edge of hepatology. Gut. 2017;66:1329-1340 pubmed publisher
    ..We discuss the development of new research tools and animal models as well as potential clinical applications, strategies and challenges. ..
  51. Cuculis L, Schroeder C. A Single-Molecule View of Genome Editing Proteins: Biophysical Mechanisms for TALEs and CRISPR/Cas9. Annu Rev Chem Biomol Eng. 2017;8:577-597 pubmed publisher
    ..These technologies have facilitated a striking number of gene editing applications in a variety of organisms; however, we are only beginning to understand the molecular mechanisms ..
  52. Nishiyama J, Mikuni T, Yasuda R. Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain. Neuron. 2017;96:755-768.e5 pubmed publisher
    ..Thus, AAV- and CRISPR-Cas9-mediated HDR will be broadly useful for precise genome editing in basic and translational neuroscience. ..
  53. Li P, Shi M, Shen W, Zhang Z, Xie D, Zhang X, et al. Coordinated regulation of IFITM1, 2 and 3 genes by an IFN-responsive enhancer through long-range chromatin interactions. Biochim Biophys Acta. 2017;1860:885-893 pubmed publisher
    ..These findings expand our understanding of the mechanisms underlying the transcriptional regulation of IFITM1, 2 and 3 expression and its ability to mediate IFN signaling. ..
  54. Chen Y, Yu J, Niu Y, Qin D, Liu H, Li G, et al. Modeling Rett Syndrome Using TALEN-Edited MECP2 Mutant Cynomolgus Monkeys. Cell. 2017;169:945-955.e10 pubmed publisher
  55. Sasai M, Sakaguchi N, Ma J, Nakamura S, Kawabata T, Bando H, et al. Essential role for GABARAP autophagy proteins in interferon-inducible GTPase-mediated host defense. Nat Immunol. 2017;18:899-910 pubmed publisher
    ..Thus, GABARAPs are uniquely required for antimicrobial host defense through cytosolic distribution of interferon-inducible GTPases. ..
  56. Carlson D, Lancto C, Zang B, Kim E, Walton M, Oldeschulte D, et al. Production of hornless dairy cattle from genome-edited cell lines. Nat Biotechnol. 2016;34:479-81 pubmed publisher
  57. Nguyen T, Anegon I. Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success. EMBO Mol Med. 2016;8:439-41 pubmed publisher
  58. Niu D, Wei H, Lin L, George H, Wang T, Lee I, et al. Inactivation of porcine endogenous retrovirus in pigs using CRISPR-Cas9. Science. 2017;357:1303-1307 pubmed publisher
    ..Our study highlights the value of PERV inactivation to prevent cross-species viral transmission and demonstrates the successful production of PERV-inactivated animals to address the safety concern in clinical xenotransplantation. ..
  59. Murovec J, Pirc Ž, Yang B. New variants of CRISPR RNA-guided genome editing enzymes. Plant Biotechnol J. 2017;15:917-926 pubmed publisher
    ..In addition, we discuss new possibilities they offer in plant biotechnology. ..
  60. Abramowski Mock U, Delhove J, Qasim W. Gene Modified T Cell Therapies for Hematological Malignancies. Hematol Oncol Clin North Am. 2017;31:913-926 pubmed publisher
    ..Recent data from clinical trials are reviewed, and an overview is provided of current and emerging manufacturing processes; consideration is also given to new developments in the pipeline. ..
  61. Fogarty N, McCarthy A, Snijders K, Powell B, Kubikova N, Blakeley P, et al. Genome editing reveals a role for OCT4 in human embryogenesis. Nature. 2017;550:67-73 pubmed publisher
    ..We conclude that CRISPR-Cas9-mediated genome editing is a powerful method for investigating gene function in the context of human development. ..
  62. Li S, Yang Y, Qiu Y, Chen Y, Xu L, Ding Q. Applications of genome editing tools in precision medicine research. Yi Chuan. 2017;39:177-188 pubmed publisher
    ..We also highlight some of the existing limitations or challenges as well as future directions. ..
  63. Ferreira R, Gatto F, Nielsen J. Exploiting off-targeting in guide-RNAs for CRISPR systems for simultaneous editing of multiple genes. FEBS Lett. 2017;591:3288-3295 pubmed publisher
  64. Hirosawa M, Fujita Y, Parr C, Hayashi K, Kashida S, Hotta A, et al. Cell-type-specific genome editing with a microRNA-responsive CRISPR-Cas9 switch. Nucleic Acids Res. 2017;45:e118 pubmed publisher
    ..Our miR-Cas9 switch system provides a promising framework for cell-type selective genome editing and cell engineering based on intracellular miRNA information. ..
  65. Dorighi K, Swigut T, Henriques T, Bhanu N, Scruggs B, Nady N, et al. Mll3 and Mll4 Facilitate Enhancer RNA Synthesis and Transcription from Promoters Independently of H3K4 Monomethylation. Mol Cell. 2017;66:568-576.e4 pubmed publisher
    ..Altogether, our results suggest that enhancer H3K4me1 provides only a minor contribution to the long-range coactivator function of Mll3/4. ..
  66. Okada M, Kanamori M, Someya K, Nakatsukasa H, Yoshimura A. Stabilization of Foxp3 expression by CRISPR-dCas9-based epigenome editing in mouse primary T cells. Epigenetics Chromatin. 2017;10:24 pubmed publisher
  67. Zuo Y, Wang H, Xu Y, Huang J, Wu S, Wu Y, et al. CRISPR/Cas9 mediated G4946E substitution in the ryanodine receptor of Spodoptera exigua confers high levels of resistance to diamide insecticides. Insect Biochem Mol Biol. 2017;89:79-85 pubmed publisher
  68. Jensen I, Inui K, Drakulić S, Jayaprakash S, Sander B, Golas M. Expression of Flp Protein in a Baculovirus/Insect Cell System for Biotechnological Applications. Protein J. 2017;36:332-342 pubmed publisher
    ..Thus, these proteins can be used for applications such as DNA-binding assays, in vitro recombination, or genome engineering. ..
  69. Hu X, Wang C, Fu Y, Liu Q, Jiao X, Wang K. Expanding the Range of CRISPR/Cas9 Genome Editing in Rice. Mol Plant. 2016;9:943-5 pubmed publisher
  70. Chen Y, Zeng S, Hu R, Wang X, Huang W, Liu J, et al. Using local chromatin structure to improve CRISPR/Cas9 efficiency in zebrafish. PLoS ONE. 2017;12:e0182528 pubmed publisher
    ..We constructed an online database for zebrafish gRNA selection based on local chromatin structure features that could prove beneficial to zebrafish homozygous mutant construction via CRISPR/Cas9. ..
  71. El Refaey M, Xu L, Gao Y, Canan B, Adesanya T, Warner S, et al. In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice. Circ Res. 2017;121:923-929 pubmed publisher
    ..This study provides evidence for using CRISPR-based genome editing as a potential therapeutic approach for restoring dystrophic cardiomyopathy structurally and functionally. ..
  72. Hsu S, Gilgenast T, Bartman C, Edwards C, Stonestrom A, Huang P, et al. The BET Protein BRD2 Cooperates with CTCF to Enforce Transcriptional and Architectural Boundaries. Mol Cell. 2017;66:102-116.e7 pubmed publisher
    ..These findings indicate that BRD2 supports boundary activity, and they raise the possibility that pharmacologic BET inhibitors can influence gene expression in part by perturbing domain boundary function. ..
  73. Wu J, Wang P, Li L, Williams N, Ji D, Zahurancik W, et al. Replication studies of carboxymethylated DNA lesions in human cells. Nucleic Acids Res. 2017;45:7276-7284 pubmed publisher
  74. Hachem A, Godwin J, Ruas M, Lee H, Ferrer Buitrago M, Ardestani G, et al. PLC? is the physiological trigger of the Ca2+ oscillations that induce embryogenesis in mammals but conception can occur in its absence. Development. 2017;144:2914-2924 pubmed publisher
    ..PLC?-null sperm now make it possible to resolve long-standing questions in fertilization biology, and to test the efficacy and safety of procedures used to treat human infertility. ..
  75. Khin N, Lowe J, Jensen L, Burgio G. No evidence for genome editing in mouse zygotes and HEK293T human cell line using the DNA-guided Natronobacterium gregoryi Argonaute (NgAgo). PLoS ONE. 2017;12:e0178768 pubmed publisher
    ..Together our findings indicate that we failed to edit using NgAgo. ..
  76. Wadhwa R, Wang X, Baladandayuthapani V, Liu B, Shiozaki H, Shimodaira Y, et al. Nuclear expression of Gli-1 is predictive of pathologic complete response to chemoradiation in trimodality treated oesophageal cancer patients. Br J Cancer. 2017;117:648-655 pubmed publisher
    ..Our validated data in OC show that nuclear Gli-1 LIs are predictive of pathCR after chemoradiation with desirable sensitivity and specificity. ..
  77. Schwartz C, Frogue K, Ramesh A, Misa J, Wheeldon I. CRISPRi repression of nonhomologous end-joining for enhanced genome engineering via homologous recombination in Yarrowia lipolytica. Biotechnol Bioeng. 2017;114:2896-2906 pubmed publisher
    ..The developed CRISPRi system enables enhanced HR in Y. lipolytica without permanent genetic knockouts and promises to be a potent tool for other metabolic engineering, synthetic biology, and functional genomics studies. ..
  78. Huang X, Zhou G, Wu W, Duan Y, Ma G, Song J, et al. Genome editing abrogates angiogenesis in vivo. Nat Commun. 2017;8:112 pubmed publisher
    ..Abnormal angiogenesis causes many ocular diseases. Here the authors employ CRISPR/Cas9 gene editing technology to silence VEGFR2, a major regulator of angiogenesis, in retinal endothelium and abrogate ..
  79. Kunzelmann S, Forstemann K. Reversible perturbations of gene regulation after genome editing in Drosophila cells. PLoS ONE. 2017;12:e0180135 pubmed publisher
  80. Butler J, Santos R, Martens G, Ladowski J, Wang Z, Li P, et al. Efficient generation of targeted and controlled mutational events in porcine cells using nuclease-directed homologous recombination. J Surg Res. 2017;212:238-245 pubmed publisher
    ..Together these strategies may be used to efficiently control mutational events. This system may be used to better use the potential of nuclease-mediated genomic editing. ..
  81. Huai C, Jia C, Sun R, Xu P, Min T, Wang Q, et al. CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice. Hum Genet. 2017;136:875-883 pubmed publisher
    ..Our study strongly supports that CRISPR/Cas9-mediated genome editing is feasible in gene therapy of genetic disorders. ..
  82. Kim S, Matsumoto T, Kagawa H, Nakamura M, Hirohata R, Ueno A, et al. Microhomology-assisted scarless genome editing in human iPSCs. Nat Commun. 2018;9:939 pubmed publisher
    ..Nevertheless, integrated selection markers obstruct scarless transgene-free gene editing. Here, we present a method for scarless selection marker excision using engineered microhomology-mediated end ..
  83. Yang H, Patel D. Inhibition Mechanism of an Anti-CRISPR Suppressor AcrIIA4 Targeting SpyCas9. Mol Cell. 2017;67:117-127.e5 pubmed publisher
    ..Our studies provide insights into anti-CRISPR-mediated suppression mechanisms for inactivating SpyCas9, thereby broadening the applicability of CRISPR-Cas regulatory tools for genome editing. ..
  84. Oike A, Kodama M, Yasumasu S, Yamamoto T, Nakamura Y, Ito E, et al. Participation of androgen and its receptor in sex determination of an amphibian species. PLoS ONE. 2017;12:e0178067 pubmed publisher
    ..These results suggest that AR with its androgen ligand functions as a male sex-determinant in the ZW type R. rugosa frogs. ..
  85. Yin K, Gao C, Qiu J. Progress and prospects in plant genome editing. Nat Plants. 2017;3:17107 pubmed publisher
    ..However, it would be highly desirable to perform precision gene editing in plants, especially in transformation-recalcitrant species...
  86. Rahman M, Rahman M. CRISPRpred: A flexible and efficient tool for sgRNAs on-target activity prediction in CRISPR/Cas9 systems. PLoS ONE. 2017;12:e0181943 pubmed publisher
    ..CRISPRpred is enough flexible to extract relevant features and use them in a learning algorithm. The source code of our entire software with relevant dataset can be found in the following link: https://github.com/khaled-buet/CRISPRpred. ..
  87. Ryu Y, Chandran S, Kim K, Lee S. Oligo- and dsDNA-mediated genome editing using a tetA dual selection system in Escherichia coli. PLoS ONE. 2017;12:e0181501 pubmed publisher
    ..Thus, this method could be used to achieve scarless, proficient, and targeted genome editing for engineering E. coli strains. ..
  88. Rogers G, Cannon P. Gene Therapy Approaches to Human Immunodeficiency Virus and Other Infectious Diseases. Hematol Oncol Clin North Am. 2017;31:883-895 pubmed publisher
    Advances in gene therapy technologies, particularly in gene editing, are suggesting new avenues for the treatment of human immunodeficiency virus and other infectious diseases...
  89. Maxwell K. The Anti-CRISPR Story: A Battle for Survival. Mol Cell. 2017;68:8-14 pubmed publisher
    ..decade has seen the fields of molecular biology and genetics transformed by the development of CRISPR-based gene editing technologies...
  90. Ukai H, Kiyonari H, Ueda H. Production of knock-in mice in a single generation from embryonic stem cells. Nat Protoc. 2017;12:2513-2530 pubmed publisher
    ..In this report, we describe the basic technologies and protocols for this procedure, and discuss the current challenges for next-generation mammalian genetics in organism-level systems biology studies...