The promise and challenge of therapeutic genome editing

References 116

1. 1.

   Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA & Charpentier E. A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity. Science 337, 816–821 (2012 ).

   Not in our library10.1126/science.1225829

2. 2.

   Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, Hsu PD, Wu X, Jiang W, Marraffini LA & Zhang F. Multiplex genome engineering using CRISPR/Cas systems. Science 339, 819–823 (2013 ).

   Not in our library10.1126/science.1231143

3. 3.

   Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, Norville JE & Church GM RNA-guided human genome engineering via Cas9. Science 339, 823–826 (2013 ).

   Not in our library10.1126/science.1232033

4. 4.
   RNA-programmed genome editing in human cells · 2013

   No notice
5. 5.

   Cho SW, Kim S, Kim JM & Kim J-S Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat. Biotechnol 31, 230–232 (2013 ).

   Not in our library10.1038/nbt.2507

6. 6.

   Knott GJ & Doudna JA CRISPR-Cas guides the future of genetic engineering. Science 361, 866–869 (2018 ).

   Not in our library10.1126/science.aat5011

7. 7.

   Hidalgo-Cantabrana C, Goh YJ & Barrangou R. Characterization and Repurposing of Type I and Type II CRISPR-Cas Systems in Bacteria. J. Mol. Biol 431, 21–33 (2019 ).

   Not in our library10.1016/j.jmb.2018.09.013

8. 8.

   Bao A, Burritt DJ, Chen H, Zhou X, Cao D. & Tran L-SP The CRISPR/Cas9 system and its applications in crop genome editing. Crit. Rev. Biotechnol 39, 321–336 (2019 ).

   Not in our library10.1080/07388551.2018.1554621

9. 9.

   Terns MP CRISPR-Based Technologies: Impact of RNA-Targeting Systems. Mol. Cell 72, 404–412 (2018 ).

   Not in our library10.1016/j.molcel.2018.09.018

10. 10.

    High KA & Roncarolo MG Gene Therapy. N. Engl. J. Med 381, 455–464 (2019 ).

    Not in our library10.1056/nejmra1706910

11. 11.

    Pauling L. & Itano HA Sickle cell anemia a molecular disease. Science 110, 543–548 (1949 ).

    Not in our library10.1126/science.110.2865.543

12. 12.

    Ingram VM Gene Mutations in Human Hæmoglobin: the Chemical Difference Between Normal and Sickle Cell Hæmoglobin. Nature 180, 326–328 (1957 ).

    Not in our library10.1038/180326a0

13. 13.

    Shieh PB Emerging Strategies in the Treatment of Duchenne Muscular Dystrophy. Neurotherapeutics 15, 840–848 (2018 ).

    Not in our library10.1007/s13311-018-00687-z

14. 14.

    Min Y-L, Bassel-Duby R. & Olson EN CRISPR Correction of Duchenne Muscular Dystrophy. Annu. Rev. Med 70, 239–255 (2019 ).

    Not in our library10.1146/annurev-med-081117-010451

15. 15.

    Long C, McAnally JR, Shelton JM, Mireault AA, Bassel-Duby R. & Olson EN Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science 345, 1184–1188 (2014 ).

    Not in our library10.1126/science.1254445

16. 16.

    Miller JC, Tan S, Qiao G, Barlow KA, Wang J, Xia DF, Meng X, Paschon DE, Leung E, Hinkley SJ, Dulay GP, Hua KL, Ankoudinova I, Cost GJ, Urnov FD, Zhang HS, Holmes MC, Zhang L, Gregory PD & Rebar EJ A TALE nuclease architecture for efficient genome editing. Nat. Biotechnol 29, 143–148 (2011 ).

    Not in our library10.1038/nbt.1755

17. 17.

    Joglekar AV, Gray D, Kaufman ML, Urbinati F, Senadheera S, Cost G, Reik A, Holmes MC, Gregory PD, Hollis RP & Kohn DB Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34+ Cells. Blood 122, 2904–2904 (2013 ).

    Not in our library

18. 18.
    Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells · 2017

    Not checked
19. 19.

    Qasim W, Zhan H, Samarasinghe S, Adams S, Amrolia P, Stafford S, Butler K, Rivat C, Wright G, Somana K, Ghorashian S, Pinner D, Ahsan G, Gilmour K, Lucchini G, Inglott S, Mifsud W, Chiesa R, Peggs KS, Chan L, Farzeneh F, Thrasher AJ, Vora A, Pule M. & Veys P. Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells. Sci. Transl. Med 9, 1–9 (2017 ).

    Not in our library10.1126/scitranslmed.aaj2013

20. 20.

    Gasiunas G, Barrangou R, Horvath P. & Siksnys V. Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria. Proc. Natl. Acad. Sci. U. S. A 109, E2579–86 (2012 ).

    Not in our library10.1073/pnas.1208507109

21. 21.

    Szostak JW, Orr-Weaver TL, Rothstein RJ & Stahl FW The double-strand-break repair model for recombination. Cell 33, 25–35 (1983 ).

    Not in our library10.1016/0092-8674(83)90331-8

22. 22.

    Stark JM, Pierce AJ, Oh J, Pastink A. & Jasin M. Genetic steps of mammalian homologous repair with distinct mutagenic consequences. Mol. Cell. Biol 24, 9305–9316 (2004 ).

    Not in our library10.1128/mcb.24.21.9305-9316.2004

23. 23.

    Liu J-J, Orlova N, Oakes BL, Ma E, Spinner HB, Baney KLM, Chuck J, Tan D, Knott GJ, Harrington LB, Al-Shayeb B, Wagner A, Brötzmann J, Staahl BT, Taylor KL, Desmarais J, Nogales E. & Doudna JA CasX enzymes comprise a distinct family of RNA-guided genome editors. Nature 566, 218–223 (2019 ).

    Not in our library10.1038/s41586-019-0908-x

24. 24.

    Yan WX, Chong S, Zhang H, Makarova KS, Koonin EV, Cheng DR & Scott DA Cas13d Is a Compact RNA-Targeting Type VI CRISPR Effector Positively Modulated by a WYL-Domain-Containing Accessory Protein. Mol. Cell 70, 327–339.e5 (2018 ).

    Not in our library10.1016/j.molcel.2018.02.028

25. 25.

    Komor AC, Kim YB, Packer MS, Zuris JA & Liu DR Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. Nature 533, 420–424 (2016 ).

    Not in our library10.1038/nature17946

26. 26.

    Nishida K, Arazoe T, Yachie N, Banno S, Kakimoto M, Tabata M, Mochizuki M, Miyabe A, Araki M, Hara KY, Shimatani Z. & Kondo A. Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems. Science 353, (2016 ).

    Not in our library10.1126/science.aaf8729

27. 27.

    Sharon E, Chen S-AA, Khosla NM, Smith JD, Pritchard JK & Fraser HB Functional Genetic Variants Revealed by Massively Parallel Precise Genome Editing. Cell 175, 544–557.e16 (2018 ).

    Not in our library10.1016/j.cell.2018.08.057

28. 28.

    Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A. & Liu DR Search-and-replace genome editing without double-strand breaks or donor DNA. Nature (2019 )

    Not in our library10.1038/s41586-019-1711-4

29. 29.

    Qi LS, Larson MH, Gilbert LA, Doudna JA, Weissman JS, Arkin AP & Lim WA Repurposing CRISPR as an RNA-guided platform for sequence-specific control of gene expression. Cell 152, 1173–1183 (2013 ).

    Not in our library10.1016/j.cell.2013.02.022

30. 30.

    Gilbert LA, Horlbeck MA, Adamson B, Villalta JE, Chen Y, Whitehead EH, Guimaraes C, Panning B, Ploegh HL, Bassik MC, Qi LS, Kampmann M. & Weissman JS Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation. Cell 159, 647–661 (2014 ).

    Not in our library10.1016/j.cell.2014.09.029

31. 31.

    Pickar-Oliver A. & Gersbach CA The next generation of CRISPR-Cas technologies and applications. Nat. Rev. Mol. Cell Biol 20, 490–507 (2019 ).

    Not in our library10.1038/s41580-019-0131-5

32. 32.

    Xu X. & Qi LS A CRISPR-dCas Toolbox for Genetic Engineering and Synthetic Biology. J. Mol. Biol 431, 34–47 (2019 ).

    Not in our library10.1016/j.jmb.2018.06.037

33. 33.

    Grünewald J, Zhou R, Garcia SP, Iyer S, Lareau CA, Aryee MJ & Joung JK Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors. Nature 569, 433–437 (2019 ).

    Not in our library10.1038/s41586-019-1161-z

34. 34.

    Zhou C, Sun Y, Yan R, Liu Y, Zuo E, Gu C, Han L, Wei Y, Hu X, Zeng R, Li Y, Zhou H, Guo F. & Yang H. Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis. Nature 571, 275–278 (2019 ).

    Not in our library10.1038/s41586-019-1314-0

35. 35.

    Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F. & Miccio A. Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. Blood 131, 1960–1973 (2018 ).

    Not in our library10.1182/blood-2017-10-811505

36. 36.

    Chung JE, Magis W, Vu J, Heo S-J, Wartiovaara K, Walters MC, Kurita R, Nakamura Y, Boffelli D, Martin DIK, Corn JE & DeWitt MA CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells. PLoS One 14, e0208237 (2019 ).

    Not in our library10.1371/journal.pone.0208237

37. 37.

    Bjurström CF, Mojadidi M, Phillips J, Kuo C, Lai S, Lill GR, Cooper A, Kaufman M, Urbinati F, Wang X, Hollis RP & Kohn DB Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases. Mol. Ther. Nucleic Acids 5, e351 (2016 ).

    Not in our library10.1038/mtna.2016.52

38. 38.

    Liu N, Hargreaves VV, Zhu Q, Kurland JV, Hong J, Kim W, Sher F, MaciasTrevino C, Rogers JM, Kurita R, Nakamura Y, Yuan G-C, Bauer DE, Xu J, Bulyk ML & Orkin SH Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch. Cell 173, 430–442.e17 (2018 ).

    Not in our library10.1016/j.cell.2018.03.016

39. 39.

    Shariati L, Khanahmad H, Salehi M, Hejazi Z, Rahimmanesh I, Tabatabaiefar MA & Modarressi MH Genetic disruption of the KLF1 gene to overexpress the γ-globin gene using the CRISPR/Cas9 system. J. Gene Med 18, 294–301 (2016 ).

    Not in our library10.1002/jgm.2928

40. 40.

    Martyn GE, Wienert B, Yang L, Shah M, Norton LJ, Burdach J, Kurita R, Nakamura Y, Pearson RCM, Funnell APW, Quinlan KGR & Crossley M. Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding. Nat. Genet 50, 498–503 (2018 ).

    Not in our library10.1038/s41588-018-0085-0

41. 41.

    Grevet JD, Lan X, Hamagami N, Edwards CR, Sankaranarayanan L, Ji X, Bhardwaj SK, Face CJ, Posocco DF, Abdulmalik O, Keller CA, Giardine B, Sidoli S, Garcia BA, Chou ST, Liebhaber SA, Hardison RC, Shi J. & Blobel GA Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cells. Science 361, 285–290 (2018 ).

    Not in our library10.1126/science.aao0932

42. 42.

    Martyn GE, Wienert B, Kurita R, Nakamura Y, Quinlan KGR & Crossley M. A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site. Blood 133, 852–856 (2019 ).

    Not in our library10.1182/blood-2018-07-863951

43. 43.

    Lomova A, Clark DN, Campo-Fernandez B, Flores-Bjurström C, Kaufman ML, Fitz-Gibbon S, Wang X, Miyahira EY, Brown D, DeWitt MA, Corn JE, Hollis RP, Romero Z. & Kohn DB Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells 37, 284–294 (2019 ).

    Not in our library10.1002/stem.2935

44. 44.

    Schumann K, Lin S, Boyer E, Simeonov DR, Subramaniam M, Gate RE, Haliburton GE, Ye CJ, Bluestone JA, Doudna JA & Marson A. Generation of knock-in primary human T cells using Cas9 ribonucleoproteins. Proc. Natl. Acad. Sci. U. S. A 112, 10437–10442 (2015 ).

    Not in our library10.1073/pnas.1512503112

45. 45.

    Dever DP, Bak RO, Reinisch A, Camarena J, Washington G, Nicolas CE, Pavel-Dinu M, Saxena N, Wilkens AB, Mantri S, Uchida N, Hendel A, Narla A, Majeti R, Weinberg KI & Porteus MH CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells. Nature 539, 384–389 (2016 ).

    Not in our library10.1038/nature20134

46. 46.

    DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo S-J, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DIK & Corn JE Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. Sci. Transl. Med 8, 360ra134 (2016 ).

    Not in our library10.1126/scitranslmed.aaf9336

47. 47.

    Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA & Bauer DE Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat. Med (2019 )

    Not in our library10.1038/s41591-019-0401-y

48. 48.

    Amoasii L, Long C, Li H, Mireault AA, Shelton JM, Sanchez-Ortiz E, McAnally JR, Bhattacharyya S, Schmidt F, Grimm D, Hauschka SD, Bassel-Duby R. & Olson EN Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. Sci. Transl. Med 9, (2017 ).

    Not in our library10.1126/scitranslmed.aan8081

49. 49.

    Gaudelli NM, Komor AC, Rees HA, Packer MS, Badran AH, Bryson DI & Liu DR Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage. Nature 551, 464–471 (2017 ).

    Not in our library10.1038/nature24644

50. 50.

    Amoasii L, Hildyard JCW, Li H, Sanchez-Ortiz E, Mireault A, Caballero D, Harron R, Stathopoulou T-R, Massey C, Shelton JM, Bassel-Duby R, Piercy RJ & Olson EN Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Science 362, 86–91 (2018 ).

    Not in our library10.1126/science.aau1549

51. 51.

    Zuo E, Sun Y, Wei W, Yuan T, Ying W, Sun H, Yuan L, Steinmetz LM, Li Y. & Yang H. Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos. Science 364, 289–292 (2019 ).

    Not in our library10.1126/science.aav9973

52. 52.

    Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ & High KA In vivo genome editing of the albumin locus as a platform for protein replacement therapy. Blood 126, 1777–1784 (2015 ).

    Not in our library10.1182/blood-2014-12-615492

53. 53.

    Laoharawee K, DeKelver RC, Podetz-Pedersen KM, Rohde M, Sproul S, Nguyen H-O, Nguyen T, St Martin SJ, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T. & McIvor RS Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing. Mol. Ther 26, 1127–1136 (2018 ).

    Not in our library10.1016/j.ymthe.2018.03.002

54. 54.

    Maeder ML, Stefanidakis M, Wilson CJ, Baral R, Barrera LA, Bounoutas GS, Bumcrot D, Chao H, Ciulla DM, DaSilva JA, Dass A, Dhanapal V, Fennell TJ, Friedland AE, Giannoukos G, Gloskowski SW, Glucksmann A, Gotta GM, Jayaram H, Haskett SJ, Hopkins B, Horng JE, Joshi S, Marco E, Mepani R, Reyon D, Ta T, Tabbaa DG, Samuelsson SJ, Shen S, Skor MN, Stetkiewicz P, Wang T, Yudkoff C, Myer VE, Albright CF & Jiang H. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10. Nat. Med 25, 229–233 (2019 ).

    Not in our library10.1038/s41591-018-0327-9

55. 55.

    Finn JD, Smith AR, Patel MC, Shaw L, Youniss MR, van Heteren J, Dirstine T, Ciullo C, Lescarbeau R, Seitzer J, Shah RR, Shah A, Ling D, Growe J, Pink M, Rohde E, Wood KM, Salomon WE, Harrington WF, Dombrowski C, Strapps WR, Chang Y. & Morrissey DV A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing. Cell Rep. 22, 2227–2235 (2018 ).

    Not in our library10.1016/j.celrep.2018.02.014

56. 56.

    Hultquist JF, Schumann K, Woo JM, Manganaro L, McGregor MJ, Doudna J, Simon V, Krogan NJ & Marson A. A Cas9 Ribonucleoprotein Platform for Functional Genetic Studies of HIV-Host Interactions in Primary Human T Cells. Cell Rep. 17, 1438–1452 (2016 ).

    Not in our library10.1016/j.celrep.2016.09.080

57. 57.

    Wang J-Z, Wu P, Shi Z-M, Xu Y-L & Liu Z-J The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD. Brain Dev. 39, 547–556 (2017 ).

    Not in our library10.1016/j.braindev.2017.03.024

58. 58.

    Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L’Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM & Kaspar BK Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. N. Engl. J. Med 377, 1713–1722 (2017 ).

    Not in our library10.1056/nejmoa1706198

59. 59.

    Yang Y, Wang L, Bell P, McMenamin D, He Z, White J, Yu H, Xu C, Morizono H, Musunuru K, Batshaw ML & Wilson JM A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice. Nat. Biotechnol 34, 334–338 (2016 ).

    Not in our library10.1038/nbt.3469

60. 60.
    In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease · 2017

    No notice
61. 61.
    Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy · 2017

    Corrected
62. 62.

    Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D. & Gersbach CA In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351, 403–407 (2016 ).

    Not in our library10.1126/science.aad5143

63. 63.

    Tabebordbar M, Zhu K, Cheng JKW, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM & Wagers AJ In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351, 407–411 (2016 ).

    Not in our library10.1126/science.aad5177

64. 64.

    Li H, Sheng C, Liu H, Wang S, Zhao J, Yang L, Jia L, Li P, Wang L, Xie J, Xu D, Sun Y, Qiu S. & Song H. Inhibition of HBV Expression in HBV Transgenic Mice Using AAV-Delivered CRISPR-SaCas9. Front. Immunol 9, 2080 (2018 ).

    Not in our library10.3389/fimmu.2018.02080

65. 65.
    In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni · 2017

    No notice
66. 66.

    Charlesworth CT, Deshpande PS, Dever DP, Dejene B, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Camarena J, Weinberg KI & Porteus MH Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans. bioRxiv 243345 (2018 )

    Not in our library10.1101/243345

67. 67.
    Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population · 2018

    No notice
68. 68.

    Sandoval IM, Kuhn NM & Manfredsson FP Multimodal Production of Adeno-Associated Virus. Methods Mol. Biol 1937, 101–124 (2019 ).

    Not in our library10.1007/978-1-4939-9065-8_6

69. 69.

    Sandro Q, Relizani K. & Benchaouir R. AAV Production Using Baculovirus Expression Vector System. Methods Mol. Biol 1937, 91–99 (2019 ).

    Not in our library10.1007/978-1-4939-9065-8_5

70. 70.

    Strobel B, Zuckschwerdt K, Zimmermann G, Mayer C, Eytner R, Rechtsteiner P, Kreuz S. & Lamla T. Standardized, Scalable, and Timely Flexible Adeno-Associated Virus Vector Production Using Frozen High-Density HEK-293 Cell Stocks and CELLdiscs. Hum. Gene Ther. Methods 30, 23–33 (2019 ).

    Not in our library10.1089/hgtb.2018.228

71. 71.

    Miller JB, Zhang S, Kos P, Xiong H, Zhou K, Perelman SS, Zhu H. & Siegwart DJ Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA. Angew. Chem. Int. Ed Engl 56, 1059–1063 (2017 ).

    Not in our library10.1002/anie.201610209

72. 72.

    Wang M, Zuris JA, Meng F, Rees H, Sun S, Deng P, Han Y, Gao X, Pouli D, Wu Q, Georgakoudi I, Liu DR & Xu Q. Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles. Proc. Natl. Acad. Sci. U. S. A 113, 2868–2873 (2016 ).

    Not in our library10.1073/pnas.1520244113

73. 73.
    In vivo base editing of post-mitotic sensory cells · 2018

    No notice
74. 74.

    Gao X, Tao Y, Lamas V, Huang M, Yeh W-H, Pan B, Hu Y-J, Hu JH, Thompson DB, Shu Y, Li Y, Wang H, Yang S, Xu Q, Polley DB, Liberman MC, Kong W-J, Holt JR, Chen Z-Y & Liu DR Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents. Nature 553, 217–221 (2018 ).

    Not in our library10.1038/nature25164

75. 75.

    Zuris JA, Thompson DB, Shu Y, Guilinger JP, Bessen JL, Hu JH, Maeder ML, Joung JK, Chen Z-Y & Liu DR Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo. Nat. Biotechnol 33, 73–80 (2015 ).

    Not in our library10.1038/nbt.3081

76. 76.

    Staahl BT, Benekareddy M, Coulon-Bainier C, Banfal AA, Floor SN, Sabo JK, Urnes C, Munares GA, Ghosh A. & Doudna JA Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes. Nat. Biotechnol 35, 431–434 (2017 ).

    Not in our library10.1038/nbt.3806

77. 77.

    Ding Y, Jiang Z, Saha K, Kim CS, Kim ST, Landis RF & Rotello VM Gold nanoparticles for nucleic acid delivery. Mol. Ther 22, 1075–1083 (2014 ).

    Not in our library10.1038/mt.2014.30

78. 78.

    Glass Z, Li Y. & Xu Q. Nanoparticles for CRISPR-Cas9 delivery. Nat Biomed Eng 1, 854–855 (2017 ).

    Not in our library10.1038/s41551-017-0158-x

79. 79.

    Kawahata Y, Takatsuto S, Ikekawa N, Murata M. & Omura S. Synthesis of a new amino acid-antibiotic, oxetin and its three stereoisomers. Chem. Pharm. Bull 34, 3102–3110 (1986 ).

    Not in our library10.1248/cpb.34.3102

80. 80.

    Lee K, Conboy M, Park HM, Jiang F, Kim HJ, Dewitt MA, Mackley VA, Chang K, Rao A, Skinner C, Shobha T, Mehdipour M, Liu H, Huang W-C, Lan F, Bray NL, Li S, Corn JE, Kataoka K, Doudna JA, Conboy I. & Murthy N. Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair. Nat Biomed Eng 1, 889–901 (2017 ).

    Not in our library10.1038/s41551-017-0137-2

81. 81.

    Sago CD, Lokugamage MP, Paunovska K, Vanover DA, Monaco CM, Shah NN, Gamboa Castro M, Anderson SE, Rudoltz TG, Lando GN, Munnilal Tiwari P, Kirschman JL, Willett N, Jang YC, Santangelo PJ, Bryksin AV & Dahlman JE High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing. Proc. Natl. Acad. Sci. U. S. A 115, E9944–E9952 (2018 ).

    Not in our library10.1073/pnas.1811276115

82. 82.

    Qin W. & Wang H. Delivery of CRISPR-Cas9 into Mouse Zygotes by Electroporation. Methods Mol. Biol 1874, 179–190 (2019 ).

    Not in our library10.1007/978-1-4939-8831-0_10

83. 83.

    Tanihara F, Hirata M, Nguyen NT, Le QA, Hirano T, Takemoto T, Nakai M, Fuchimoto D-I & Otoi T. Generation of a TP53-modified porcine cancer model by CRISPR/Cas9-mediated gene modification in porcine zygotes via electroporation. PLoS One 13, e0206360 (2018 ).

    Not in our library10.1371/journal.pone.0206360

84. 84.

    Xu L, Park KH, Zhao L, Xu J, El Refaey M, Gao Y, Zhu H, Ma J. & Han R. CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice. Mol. Ther 24, 564–569 (2016 ).

    Not in our library10.1038/mt.2015.192

85. 85.

    Kim S, Kim D, Cho SW, Kim J. & Kim J-S Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins. Genome Res. 24, 1012–1019 (2014 ).

    Not in our library10.1101/gr.171322.113

86. 86.
    Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery · 2014

    No notice
87. 87.

    Gaj T, Staahl BT, Rodrigues GMC, Limsirichai P, Ekman FK, Doudna JA & Schaffer DV Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery. Nucleic Acids Res. 45, e98 (2017 ).

    Not in our library10.1093/nar/gkx154

88. 88.

    Rouet R, Thuma BA, Roy MD, Lintner NG, Rubitski DM, Finley JE, Wisniewska HM, Mendonsa R, Hirsh A, de Oñate L, Compte Barrón J, McLellan TJ, Bellenger J, Feng X, Varghese A, Chrunyk BA, Borzilleri K, Hesp KD, Zhou K, Ma N, Tu M, Dullea R, McClure KF, Wilson RC, Liras S, Mascitti V. & Doudna JA Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell-Type-Specific Gene Editing. J. Am. Chem. Soc 140, 6596–6603 (2018 ).

    Not in our library10.1021/jacs.8b01551

89. 89.

    Yin J, Wang Q, Hou S, Bao L, Yao W. & Gao X. Potent Protein Delivery into Mammalian Cells via a Supercharged Polypeptide. J. Am. Chem. Soc 140, 17234–17240 (2018 ).

    Not in our library10.1021/jacs.8b10299

90. 90.

    Riley RS, June CH, Langer R. & Mitchell MJ Delivery technologies for cancer immunotherapy. Nat. Rev. Drug Discov 18, 175–196 (2019 ).

    Not in our library10.1038/s41573-018-0006-z

91. 91.

    Sun Y, Su J, Liu G, Chen J, Zhang X, Zhang R, Jiang M. & Qiu M. Advances of blood cell-based drug delivery systems. Eur. J. Pharm. Sci 96, 115–128 (2017 ).

    Not in our library10.1016/j.ejps.2016.07.021

92. 92.

    Sharma P, Cho HA, Lee J-W, Ham WS, Park BC, Cho N-H & Kim YK Efficient intracellular delivery of biomacromolecules employing clusters of zinc oxide nanowires. Nanoscale 9, 15371–15378 (2017 ).

    Not in our library10.1039/c7nr05219g

93. 93.

    Kim D, Luk K, Wolfe SA & Kim J-S Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases. Annu. Rev. Biochem (2019 )

    Not in our library10.1146/annurev-biochem-013118-111730

94. 94.

    Kosicki M, Tomberg K. & Bradley A. Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements. Nat. Biotechnol 36, 765–771 (2018 ).

    Not in our library10.1038/nbt.4192

95. 95.

    Poirot L, Philip B, Schiffer-Mannioui C, Le Clerre D, Chion-Sotinel I, Derniame S, Potrel P, Bas C, Lemaire L, Galetto R, Lebuhotel C, Eyquem J, Cheung GW-K, Duclert A, Gouble A, Arnould S, Peggs K, Pule M, Scharenberg AM & Smith J. Multiplex Genome-Edited T-cell Manufacturing Platform for ‘Off-the-Shelf’ Adoptive T-cell Immunotherapies. Cancer Res. 75, 3853–3864 (2015 ).

    Not in our library10.1158/0008-5472.can-14-3321

96. 96.
    Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6 · 2017

    Corrected
97. 97.

    Tichy ED, Pillai R, Deng L, Liang L, Tischfield J, Schwemberger SJ, Babcock GF & Stambrook PJ Mouse embryonic stem cells, but not somatic cells, predominantly use homologous recombination to repair double-strand DNA breaks. Stem Cells Dev. 19, 1699–1711 (2010 ).

    Not in our library10.1089/scd.2010.0058

98. 98.

    Buechele C, Breese EH, Schneidawind D, Lin C-H, Jeong J, Duque-Afonso J, Wong SHK, Smith KS, Negrin RS, Porteus M. & Cleary ML MLL leukemia induction by genome editing of human CD34+ hematopoietic cells. Blood 126, 1683–1694 (2015 ).

    Not in our library10.1182/blood-2015-05-646398

99. 99.

    Maddalo D, Manchado E, Concepcion CP, Bonetti C, Vidigal JA, Han Y-C, Ogrodowski P, Crippa A, Rekhtman N, de Stanchina E, Lowe SW & Ventura A. In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system. Nature 516, 423–427 (2014 ).

    Not in our library10.1038/nature13902

100. 100.

     Eskandari T. NIST Genome Editing Consortium. NIST at <https://www.nist.gov/programsprojects/nist-genome-editing-consortium>

     Not in our library

101. 101.

     Gilbert LA, Larson MH, Morsut L, Liu Z, Brar GA, Torres SE, Stern-Ginossar N, Brandman O, Whitehead EH, Doudna JA, Lim WA, Weissman JS & Qi LS CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell 154, 442–451 (2013 ).

     Not in our library10.1016/j.cell.2013.06.044

102. 102.

     Liu XS, Wu H, Ji X, Stelzer Y, Wu X, Czauderna S, Shu J, Dadon D, Young RA & Jaenisch R. Editing DNA Methylation in the Mammalian Genome. Cell 167, 233–247.e17 (2016 ).

     Not in our library10.1016/j.cell.2016.08.056

103. 103.

     Wilson RC & Carroll D. The Daunting Economics of Therapeutic Genome Editing. CRISPR J 2, 280–284 (2019 ).

     Not in our library10.1089/crispr.2019.0052

104. 104.

     Wagner DL, Amini L, Wendering DJ, Burkhardt L-M, Akyüz L, Reinke P, Volk H-D & Schmueck-Henneresse M. High prevalence of Streptococcus pyogenes Cas9reactive T cells within the adult human population. Nat. Med 25, 242–248 (2019 ).

     Not in our library10.1038/s41591-018-0204-6

105. 105.

     Haapaniemi E, Botla S, Persson J, Schmierer B. & Taipale J. CRISPR-Cas9 genome editing induces a p53-mediated DNA damage response. Nat. Med 24, 927–930 (2018 ).

     Not in our library10.1038/s41591-018-0049-z

106. 106.

     Ihry RJ, Worringer KA, Salick MR, Frias E, Ho D, Theriault K, Kommineni S, Chen J, Sondey M, Ye C, Randhawa R, Kulkarni T, Yang Z, McAllister G, Russ C, Reece-Hoyes J, Forrester W, Hoffman GR, Dolmetsch R. & Kaykas A. p53 inhibits CRISPR-Cas9 engineering in human pluripotent stem cells. Nat. Med 24, 939–946 (2018 ).

     Not in our library10.1038/s41591-018-0050-6

107. 107.

     Schiroli G, Conti A, Ferrari S, Della Volpe L, Jacob A, Albano L, Beretta S, Calabria A, Vavassori V, Gasparini P, Salataj E, Ndiaye-Lobry D, Brombin C, Chaumeil J, Montini E, Merelli I, Genovese P, Naldini L. & Di Micco R. Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response. Cell Stem Cell 24, 551–565.e8 (2019 ).

     Not in our library10.1016/j.stem.2019.02.019

108. 108.

     Xu L, Wang J, Liu Y, Xie L, Su B, Mou D, Wang L, Liu T, Wang X, Zhang B, Zhao L, Hu L, Ning H, Zhang Y, Deng K, Liu L, Lu X, Zhang T, Xu J, Li C, Wu H, Deng H. & Chen H. CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia. N. Engl. J. Med 381, 1240–1247 (2019 ).

     Not in our library10.1056/nejmoa1817426

109. 109.

     Porteus MH A New Class of Medicines through DNA Editing. N. Engl. J. Med 380, 947–959 (2019 ).

     Not in our library10.1056/nejmra1800729

110. 110.
     CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes · 2015

     No notice
111. 111.

     Fogarty NME, McCarthy A, Snijders KE, Powell BE, Kubikova N, Blakeley P, Lea R, Elder K, Wamaitha SE, Kim D, Maciulyte V, Kleinjung J, Kim J-S, Wells D, Vallier L, Bertero A, Turner JMA & Niakan KK Genome editing reveals a role for OCT4 in human embryogenesis. Nature 550, 67–73 (2017 ).

     Not in our library10.1038/nature24033

112. 112.

     Tang L, Zeng Y, Du H, Gong M, Peng J, Zhang B, Lei M, Zhao F, Wang W, Li X. & Liu J. CRISPR/Cas9-mediated gene editing in human zygotes using Cas9 protein. Mol. Genet. Genomics 292, 525–533 (2017 ).

     Not in our library10.1007/s00438-017-1299-z

113. 113.

     Ma H, Marti-Gutierrez N, Park S-W, Wu J, Lee Y, Suzuki K, Koski A, Ji D, Hayama T, Ahmed R, Darby H, Van Dyken C, Li Y, Kang E, Park A-R, Kim D, Kim S-T, Gong J, Gu Y, Xu X, Battaglia D, Krieg SA, Lee DM, Wu DH, Wolf DP, Heitner SB, Belmonte JCI, Amato P, Kim J-S, Kaul S. & Mitalipov S. Correction of a pathogenic gene mutation in human embryos. Nature 548, 413–419 (2017 ).

     Not in our library10.1038/nature23305

114. 114.

     Kaul S, Heitner SB & Mitalipov S. Sarcomere Gene Mutation correction. Eur. Heart J 39, 1506–1507 (2018 ).

     Not in our library10.1093/eurheartj/ehy179

115. 115.

     Egli D, Zuccaro MV, Kosicki M, Church GM, Bradley A. & Jasin M. Interhomologue repair in fertilized human eggs? Nature 560, E5–E7 (2018 ).

     Not in our library10.1038/s41586-018-0379-5

116. 116.

     Ormond KE, Mortlock DP, Scholes DT, Bombard Y, Brody LC, Faucett WA, Garrison NA, Hercher L, Isasi R, Middleton A, Musunuru K, Shriner D, Virani A. & Young CE Human Germline Genome Editing. Am. J. Hum. Genet 101, 167–176 (2017 ).

     Not in our library10.1016/j.ajhg.2017.06.012

Cited by papers we hold 13

• Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing · 2023
  No notice
• Genotoxic effects of base and prime editing in human hematopoietic stem cells · 2023
  Corrected
• Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL · 2022
  No notice
• Nonviral Delivery of CRISPR/Cas Systems in mRNA Format · 2022
  Not checked
• Structural basis for mismatch surveillance by CRISPR–Cas9 · 2022
  Corrected
• Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins · 2022
  No notice
• Evaluation of Cells and Medium Optimization for Invitro Model of Diabetic and Electrolyte Imbalance · 2021
  Not checked
• Applications, challenges, and needs for employing synthetic biology beyond the lab · 2021
  No notice
• Viral vector‐based gene therapies in the clinic · 2021
  No notice
• Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models · 2021
  No notice
• How can nanotechnology help to combat COVID-19? Opportunities and urgent need · 2020
  No notice
• DNA Damage: From Threat to Treatment · 2020
  No notice
• Methods Favoring Homology-Directed Repair Choice in Response to CRISPR/Cas9 Induced-Double Strand Breaks · 2020
  No notice

Reads

A person counts once a day per paper. We keep no cookie and no address: a reader is a hash that changes every day. Any month under five reads shows no number.