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Items: 37

1.

Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.

Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ.

Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820.

2.

Zebrafish dscaml1 Deficiency Impairs Retinal Patterning and Oculomotor Function.

Ma 马漫修 M, Ramirez AD, Wang 王彤 T, Roberts RL, Harmon KE, Schoppik D, Sharma A, Kuang C, Goei SL, Gagnon JA, Zimmerman S, Tsai SQ, Reyon D, Joung JK, Aksay ERF, Schier AF, Pan 潘於勤 YA.

J Neurosci. 2019 Nov 4. pii: 1783-19. doi: 10.1523/JNEUROSCI.1783-19.2019. [Epub ahead of print]

PMID:
31685652
3.

High levels of AAV vector integration into CRISPR-induced DNA breaks.

Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Lööv C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, György B.

Nat Commun. 2019 Sep 30;10(1):4439. doi: 10.1038/s41467-019-12449-2.

4.

Highly efficient therapeutic gene editing of human hematopoietic stem cells.

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.

Nat Med. 2019 May;25(5):776-783. doi: 10.1038/s41591-019-0401-y. Epub 2019 Mar 25.

5.

Illuminating the genome-wide activity of genome editors for safe and effective therapeutics.

Cheng Y, Tsai SQ.

Genome Biol. 2018 Dec 22;19(1):226. doi: 10.1186/s13059-018-1610-2.

6.

Defining CRISPR-Cas9 genome-wide nuclease activities with CIRCLE-seq.

Lazzarotto CR, Nguyen NT, Tang X, Malagon-Lopez J, Guo JA, Aryee MJ, Joung JK, Tsai SQ.

Nat Protoc. 2018 Nov;13(11):2615-2642. doi: 10.1038/s41596-018-0055-0.

7.

In vivo CRISPR editing with no detectable genome-wide off-target mutations.

Akcakaya P, Bobbin ML, Guo JA, Malagon-Lopez J, Clement K, Garcia SP, Fellows MD, Porritt MJ, Firth MA, Carreras A, Baccega T, Seeliger F, Bjursell M, Tsai SQ, Nguyen NT, Nitsch R, Mayr LM, Pinello L, Bohlooly-Y M, Aryee MJ, Maresca M, Joung JK.

Nature. 2018 Sep;561(7723):416-419. doi: 10.1038/s41586-018-0500-9. Epub 2018 Sep 12.

8.

Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia.

Kim MY, Yu KR, Kenderian SS, Ruella M, Chen S, Shin TH, Aljanahi AA, Schreeder D, Klichinsky M, Shestova O, Kozlowski MS, Cummins KD, Shan X, Shestov M, Bagg A, Morrissette JJD, Sekhri P, Lazzarotto CR, Calvo KR, Kuhns DB, Donahue RE, Behbehani GK, Tsai SQ, Dunbar CE, Gill S.

Cell. 2018 May 31;173(6):1439-1453.e19. doi: 10.1016/j.cell.2018.05.013. Epub 2018 May 31.

9.

Discovering the Genome-Wide Activity of CRISPR-Cas Nucleases.

Tsai SQ.

ACS Chem Biol. 2018 Feb 16;13(2):305-308. doi: 10.1021/acschembio.7b00847. Epub 2017 Dec 27.

PMID:
29281250
10.

Nodal patterning without Lefty inhibitory feedback is functional but fragile.

Rogers KW, Lord ND, Gagnon JA, Pauli A, Zimmerman S, Aksel DC, Reyon D, Tsai SQ, Joung JK, Schier AF.

Elife. 2017 Dec 7;6. pii: e28785. doi: 10.7554/eLife.28785.

11.

Towards safe therapy for immunodeficiency.

Tsai SQ.

Nat Biomed Eng. 2017 Dec;1(12):937-938. doi: 10.1038/s41551-017-0174-x. No abstract available.

PMID:
31015711
12.

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR-Cas9 nuclease off-targets.

Tsai SQ, Nguyen NT, Malagon-Lopez J, Topkar VV, Aryee MJ, Joung JK.

Nat Methods. 2017 Jun;14(6):607-614. doi: 10.1038/nmeth.4278. Epub 2017 May 1.

13.

Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells.

Kleinstiver BP, Tsai SQ, Prew MS, Nguyen NT, Welch MM, Lopez JM, McCaw ZR, Aryee MJ, Joung JK.

Nat Biotechnol. 2016 Aug;34(8):869-74. doi: 10.1038/nbt.3620. Epub 2016 Jun 27.

14.

Open-source guideseq software for analysis of GUIDE-seq data.

Tsai SQ, Topkar VV, Joung JK, Aryee MJ.

Nat Biotechnol. 2016 May 6;34(5):483. doi: 10.1038/nbt.3534. No abstract available.

PMID:
27153277
15.

Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases.

Tsai SQ, Joung JK.

Nat Rev Genet. 2016 May;17(5):300-12. doi: 10.1038/nrg.2016.28. Review.

PMID:
27087594
16.

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.

Kleinstiver BP, Pattanayak V, Prew MS, Tsai SQ, Nguyen NT, Zheng Z, Joung JK.

Nature. 2016 Jan 28;529(7587):490-5. doi: 10.1038/nature16526. Epub 2016 Jan 6.

17.

Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition.

Kleinstiver BP, Prew MS, Tsai SQ, Nguyen NT, Topkar VV, Zheng Z, Joung JK.

Nat Biotechnol. 2015 Dec;33(12):1293-1298. doi: 10.1038/nbt.3404. Epub 2015 Nov 2.

18.

Genome Editing in Human Cells Using CRISPR/Cas Nucleases.

Wyvekens N, Tsai SQ, Joung JK.

Curr Protoc Mol Biol. 2015 Oct 1;112:31.3.1-31.3.18. doi: 10.1002/0471142727.mb3103s112.

19.

Continuous directed evolution of DNA-binding proteins to improve TALEN specificity.

Hubbard BP, Badran AH, Zuris JA, Guilinger JP, Davis KM, Chen L, Tsai SQ, Sander JD, Joung JK, Liu DR.

Nat Methods. 2015 Oct;12(10):939-42. doi: 10.1038/nmeth.3515. Epub 2015 Aug 10.

20.

Engineered CRISPR-Cas9 nucleases with altered PAM specificities.

Kleinstiver BP, Prew MS, Tsai SQ, Topkar VV, Nguyen NT, Zheng Z, Gonzales AP, Li Z, Peterson RT, Yeh JR, Aryee MJ, Joung JK.

Nature. 2015 Jul 23;523(7561):481-5. doi: 10.1038/nature14592. Epub 2015 Jun 22.

21.

Dimeric CRISPR RNA-Guided FokI-dCas9 Nucleases Directed by Truncated gRNAs for Highly Specific Genome Editing.

Wyvekens N, Topkar VV, Khayter C, Joung JK, Tsai SQ.

Hum Gene Ther. 2015 Jul;26(7):425-31. doi: 10.1089/hum.2015.084.

22.

Targeted disruption of DNMT1, DNMT3A and DNMT3B in human embryonic stem cells.

Liao J, Karnik R, Gu H, Ziller MJ, Clement K, Tsankov AM, Akopian V, Gifford CA, Donaghey J, Galonska C, Pop R, Reyon D, Tsai SQ, Mallard W, Joung JK, Rinn JL, Gnirke A, Meissner A.

Nat Genet. 2015 May;47(5):469-78. doi: 10.1038/ng.3258. Epub 2015 Mar 30.

23.

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases.

Tsai SQ, Zheng Z, Nguyen NT, Liebers M, Topkar VV, Thapar V, Wyvekens N, Khayter C, Iafrate AJ, Le LP, Aryee MJ, Joung JK.

Nat Biotechnol. 2015 Feb;33(2):187-197. doi: 10.1038/nbt.3117. Epub 2014 Dec 16.

24.

Genome editing: a tool for research and therapy: towards a functional understanding of variants for molecular diagnostics using genome editing.

Tsai SQ, Iafrate AJ, Joung JK.

Nat Med. 2014 Oct;20(10):1103-4. doi: 10.1038/nm.3722. No abstract available.

PMID:
25295940
25.

Systematic screening reveals a role for BRCA1 in the response to transcription-associated DNA damage.

Hill SJ, Rolland T, Adelmant G, Xia X, Owen MS, Dricot A, Zack TI, Sahni N, Jacob Y, Hao T, McKinney KM, Clark AP, Reyon D, Tsai SQ, Joung JK, Beroukhim R, Marto JA, Vidal M, Gaudet S, Hill DE, Livingston DM.

Genes Dev. 2014 Sep 1;28(17):1957-75. doi: 10.1101/gad.241620.114.

26.

What's changed with genome editing?

Tsai SQ, Joung JK.

Cell Stem Cell. 2014 Jul 3;15(1):3-4. doi: 10.1016/j.stem.2014.06.017.

27.

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing.

Tsai SQ, Wyvekens N, Khayter C, Foden JA, Thapar V, Reyon D, Goodwin MJ, Aryee MJ, Joung JK.

Nat Biotechnol. 2014 Jun;32(6):569-76. doi: 10.1038/nbt.2908. Epub 2014 Apr 25.

28.

Broad specificity profiling of TALENs results in engineered nucleases with improved DNA-cleavage specificity.

Guilinger JP, Pattanayak V, Reyon D, Tsai SQ, Sander JD, Joung JK, Liu DR.

Nat Methods. 2014 Apr;11(4):429-35. doi: 10.1038/nmeth.2845. Epub 2014 Feb 16.

29.

Toddler: an embryonic signal that promotes cell movement via Apelin receptors.

Pauli A, Norris ML, Valen E, Chew GL, Gagnon JA, Zimmerman S, Mitchell A, Ma J, Dubrulle J, Reyon D, Tsai SQ, Joung JK, Saghatelian A, Schier AF.

Science. 2014 Feb 14;343(6172):1248636. doi: 10.1126/science.1248636. Epub 2014 Jan 9.

30.

Correction of the Crb1rd8 allele and retinal phenotype in C57BL/6N mice via TALEN-mediated homology-directed repair.

Low BE, Krebs MP, Joung JK, Tsai SQ, Nishina PM, Wiles MV.

Invest Ophthalmol Vis Sci. 2014 Jan 20;55(1):387-95. doi: 10.1167/iovs.13-13278.

31.

Targeted DNA demethylation and activation of endogenous genes using programmable TALE-TET1 fusion proteins.

Maeder ML, Angstman JF, Richardson ME, Linder SJ, Cascio VM, Tsai SQ, Ho QH, Sander JD, Reyon D, Bernstein BE, Costello JF, Wilkinson MF, Joung JK.

Nat Biotechnol. 2013 Dec;31(12):1137-42. doi: 10.1038/nbt.2726. Epub 2013 Oct 9.

32.

Engineering customized TALE nucleases (TALENs) and TALE transcription factors by fast ligation-based automatable solid-phase high-throughput (FLASH) assembly.

Reyon D, Maeder ML, Khayter C, Tsai SQ, Foley JE, Sander JD, Joung JK.

Curr Protoc Mol Biol. 2013 Jul;Chapter 12:Unit 12.16. doi: 10.1002/0471142727.mb1216s103.

33.

Differentially expressed microRNAs and affected biological pathways revealed by modulated modularity clustering (MMC) analysis of human preeclamptic and IUGR placentas.

Guo L, Tsai SQ, Hardison NE, James AH, Motsinger-Reif AA, Thames B, Stone EA, Deng C, Piedrahita JA.

Placenta. 2013 Jul;34(7):599-605. doi: 10.1016/j.placenta.2013.04.007. Epub 2013 Apr 30.

34.

Differences in X-chromosome transcriptional activity and cholesterol metabolism between placentae from swine breeds from Asian and Western origins.

Bischoff SR, Tsai SQ, Hardison NE, Motsinger-Reif AA, Freking BA, Nonneman DJ, Rohrer GA, Piedrahita JA.

PLoS One. 2013;8(1):e55345. doi: 10.1371/journal.pone.0055345. Epub 2013 Jan 31.

35.

Efficient genome editing in zebrafish using a CRISPR-Cas system.

Hwang WY, Fu Y, Reyon D, Maeder ML, Tsai SQ, Sander JD, Peterson RT, Yeh JR, Joung JK.

Nat Biotechnol. 2013 Mar;31(3):227-9. doi: 10.1038/nbt.2501. Epub 2013 Jan 29.

36.

Highly efficient generation of heritable zebrafish gene mutations using homo- and heterodimeric TALENs.

Cade L, Reyon D, Hwang WY, Tsai SQ, Patel S, Khayter C, Joung JK, Sander JD, Peterson RT, Yeh JR.

Nucleic Acids Res. 2012 Sep;40(16):8001-10. doi: 10.1093/nar/gks518. Epub 2012 Jun 7.

37.

FLASH assembly of TALENs for high-throughput genome editing.

Reyon D, Tsai SQ, Khayter C, Foden JA, Sander JD, Joung JK.

Nat Biotechnol. 2012 May;30(5):460-5. doi: 10.1038/nbt.2170.

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