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Items: 40

1.

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

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.

Nat Med. 2019 Feb;25(2):229-233. doi: 10.1038/s41591-018-0327-9. Epub 2019 Jan 21.

PMID:
30664785
2.

Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque.

McCullough KT, Boye SL, Fajardo D, Calabro K, Peterson JJ, Strang CE, Chakraborty D, Gloskowski S, Haskett S, Samuelsson S, Jiang H, Witherspoon CD, Gamlin PD, Maeder ML, Boye SE.

Hum Gene Ther. 2019 May;30(5):571-589. doi: 10.1089/hum.2018.193. Epub 2018 Dec 20.

PMID:
30358434
3.

Response to "Unexpected mutations after CRISPR-Cas9 editing in vivo".

Wilson CJ, Fennell T, Bothmer A, Maeder ML, Reyon D, Cotta-Ramusino C, Fernandez CA, Marco E, Barrera LA, Jayaram H, Albright CF, Cox GF, Church GM, Myer VE.

Nat Methods. 2018 Apr;15(4):236-237. doi: 10.1038/nmeth.4552. Epub 2018 Mar 30. No abstract available.

PMID:
29600989
4.

UDiTaS™, a genome editing detection method for indels and genome rearrangements.

Giannoukos G, Ciulla DM, Marco E, Abdulkerim HS, Barrera LA, Bothmer A, Dhanapal V, Gloskowski SW, Jayaram H, Maeder ML, Skor MN, Wang T, Myer VE, Wilson CJ.

BMC Genomics. 2018 Mar 21;19(1):212. doi: 10.1186/s12864-018-4561-9.

5.

Genome-editing Technologies for Gene and Cell Therapy.

Maeder ML, Gersbach CA.

Mol Ther. 2016 Mar;24(3):430-46. doi: 10.1038/mt.2016.10. Epub 2016 Jan 12. Review.

6.

Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications.

Friedland AE, Baral R, Singhal P, Loveluck K, Shen S, Sanchez M, Marco E, Gotta GM, Maeder ML, Kennedy EM, Kornepati AV, Sousa A, Collins MA, Jayaram H, Cullen BR, Bumcrot D.

Genome Biol. 2015 Nov 24;16:257. doi: 10.1186/s13059-015-0817-8.

7.

Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.

Gori JL, Hsu PD, Maeder ML, Shen S, Welstead GG, Bumcrot D.

Hum Gene Ther. 2015 Jul;26(7):443-51. doi: 10.1089/hum.2015.074. Review.

PMID:
26068008
8.

Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model.

Rahman SH, Kuehle J, Reimann C, Mlambo T, Alzubi J, Maeder ML, Riedel H, Fisch P, Cantz T, Rudolph C, Mussolino C, Joung JK, Schambach A, Cathomen T.

PLoS Genet. 2015 May 22;11(5):e1005239. doi: 10.1371/journal.pgen.1005239. eCollection 2015 May.

9.

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo.

Zuris JA, Thompson DB, Shu Y, Guilinger JP, Bessen JL, Hu JH, Maeder ML, Joung JK, Chen ZY, Liu DR.

Nat Biotechnol. 2015 Jan;33(1):73-80. doi: 10.1038/nbt.3081. Epub 2014 Oct 30.

10.

Targeted mutagenesis of zebrafish antithrombin III triggers disseminated intravascular coagulation and thrombosis, revealing insight into function.

Liu Y, Kretz CA, Maeder ML, Richter CE, Tsao P, Vo AH, Huarng MC, Rode T, Hu Z, Mehra R, Olson ST, Joung JK, Shavit JA.

Blood. 2014 Jul 3;124(1):142-50. doi: 10.1182/blood-2014-03-561027. Epub 2014 Apr 29.

11.

Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SOD1.

Kiskinis E, Sandoe J, Williams LA, Boulting GL, Moccia R, Wainger BJ, Han S, Peng T, Thams S, Mikkilineni S, Mellin C, Merkle FT, Davis-Dusenbery BN, Ziller M, Oakley D, Ichida J, Di Costanzo S, Atwater N, Maeder ML, Goodwin MJ, Nemesh J, Handsaker RE, Paull D, Noggle S, McCarroll SA, Joung JK, Woolf CJ, Brown RH, Eggan K.

Cell Stem Cell. 2014 Jun 5;14(6):781-95. doi: 10.1016/j.stem.2014.03.004. Epub 2014 Apr 3.

12.

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.

13.

CRISPR RNA-guided activation of endogenous human genes.

Maeder ML, Linder SJ, Cascio VM, Fu Y, Ho QH, Joung JK.

Nat Methods. 2013 Oct;10(10):977-9. doi: 10.1038/nmeth.2598. Epub 2013 Jul 25.

14.

Heritable and precise zebrafish genome editing using a CRISPR-Cas system.

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

PLoS One. 2013 Jul 9;8(7):e68708. doi: 10.1371/journal.pone.0068708. Print 2013.

15.

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.

16.

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells.

Fu Y, Foden JA, Khayter C, Maeder ML, Reyon D, Joung JK, Sander JD.

Nat Biotechnol. 2013 Sep;31(9):822-6. doi: 10.1038/nbt.2623. Epub 2013 Jun 23.

17.

TALEN-based gene correction for epidermolysis bullosa.

Osborn MJ, Starker CG, McElroy AN, Webber BR, Riddle MJ, Xia L, DeFeo AP, Gabriel R, Schmidt M, von Kalle C, Carlson DF, Maeder ML, Joung JK, Wagner JE, Voytas DF, Blazar BR, Tolar J.

Mol Ther. 2013 Jun;21(6):1151-9. doi: 10.1038/mt.2013.56. Epub 2013 Apr 2.

18.

Robust, synergistic regulation of human gene expression using TALE activators.

Maeder ML, Linder SJ, Reyon D, Angstman JF, Fu Y, Sander JD, Joung JK.

Nat Methods. 2013 Mar;10(3):243-5. doi: 10.1038/nmeth.2366. Epub 2013 Feb 10.

19.

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.

20.

Evaluation of OPEN zinc finger nucleases for direct gene targeting of the ROSA26 locus in mouse embryos.

Hermann M, Maeder ML, Rector K, Ruiz J, Becher B, Bürki K, Khayter C, Aguzzi A, Joung JK, Buch T, Pelczar P.

PLoS One. 2012;7(9):e41796. doi: 10.1371/journal.pone.0041796. Epub 2012 Sep 6.

21.

Engineering designer nucleases with customized cleavage specificities.

Sander JD, Maeder ML, Joung JK.

Curr Protoc Mol Biol. 2011 Oct;Chapter 12:Unit12.13. doi: 10.1002/0471142727.mb1213s96.

22.

In situ genetic correction of the sickle cell anemia mutation in human induced pluripotent stem cells using engineered zinc finger nucleases.

Sebastiano V, Maeder ML, Angstman JF, Haddad B, Khayter C, Yeo DT, Goodwin MJ, Hawkins JS, Ramirez CL, Batista LF, Artandi SE, Wernig M, Joung JK.

Stem Cells. 2011 Nov;29(11):1717-26. doi: 10.1002/stem.718.

23.

Induction of stable drug resistance in human breast cancer cells using a combinatorial zinc finger transcription factor library.

Lee J, Hirsh AS, Wittner BS, Maeder ML, Singavarapu R, Lang M, Janarthanan S, McDermott U, Yajnik V, Ramaswamy S, Joung JK, Sgroi DC.

PLoS One. 2011;6(7):e21112. doi: 10.1371/journal.pone.0021112. Epub 2011 Jul 19.

24.

Zinc-finger nucleases for somatic gene therapy: the next frontier.

Rahman SH, Maeder ML, Joung JK, Cathomen T.

Hum Gene Ther. 2011 Aug;22(8):925-33. doi: 10.1089/hum.2011.087. Epub 2011 Jul 22. Review.

25.

Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA).

Sander JD, Dahlborg EJ, Goodwin MJ, Cade L, Zhang F, Cifuentes D, Curtin SJ, Blackburn JS, Thibodeau-Beganny S, Qi Y, Pierick CJ, Hoffman E, Maeder ML, Khayter C, Reyon D, Dobbs D, Langenau DM, Stupar RM, Giraldez AJ, Voytas DF, Peterson RT, Yeh JR, Joung JK.

Nat Methods. 2011 Jan;8(1):67-9. doi: 10.1038/nmeth.1542. Epub 2010 Dec 12.

26.

Predicting success of oligomerized pool engineering (OPEN) for zinc finger target site sequences.

Sander JD, Reyon D, Maeder ML, Foley JE, Thibodeau-Beganny S, Li X, Regan MR, Dahlborg EJ, Goodwin MJ, Fu F, Voytas DF, Joung JK, Dobbs D.

BMC Bioinformatics. 2010 Nov 2;11:543. doi: 10.1186/1471-2105-11-543.

27.

Autonomous zinc-finger nuclease pairs for targeted chromosomal deletion.

Söllü C, Pars K, Cornu TI, Thibodeau-Beganny S, Maeder ML, Joung JK, Heilbronn R, Cathomen T.

Nucleic Acids Res. 2010 Dec;38(22):8269-76. doi: 10.1093/nar/gkq720. Epub 2010 Aug 16.

28.

Engineering single Cys2His2 zinc finger domains using a bacterial cell-based two-hybrid selection system.

Thibodeau-Beganny S, Maeder ML, Joung JK.

Methods Mol Biol. 2010;649:31-50. doi: 10.1007/978-1-60761-753-2_2.

PMID:
20680826
29.

High frequency targeted mutagenesis in Arabidopsis thaliana using zinc finger nucleases.

Zhang F, Maeder ML, Unger-Wallace E, Hoshaw JP, Reyon D, Christian M, Li X, Pierick CJ, Dobbs D, Peterson T, Joung JK, Voytas DF.

Proc Natl Acad Sci U S A. 2010 Jun 29;107(26):12028-33. doi: 10.1073/pnas.0914991107. Epub 2010 May 27.

30.

ZiFiT (Zinc Finger Targeter): an updated zinc finger engineering tool.

Sander JD, Maeder ML, Reyon D, Voytas DF, Joung JK, Dobbs D.

Nucleic Acids Res. 2010 Jul;38(Web Server issue):W462-8. doi: 10.1093/nar/gkq319. Epub 2010 Apr 30.

31.

Targeted mutagenesis in zebrafish using customized zinc-finger nucleases.

Foley JE, Maeder ML, Pearlberg J, Joung JK, Peterson RT, Yeh JR.

Nat Protoc. 2009;4(12):1855-67. doi: 10.1038/nprot.2009.209.

32.

Oligomerized pool engineering (OPEN): an 'open-source' protocol for making customized zinc-finger arrays.

Maeder ML, Thibodeau-Beganny S, Sander JD, Voytas DF, Joung JK.

Nat Protoc. 2009;4(10):1471-501. doi: 10.1038/nprot.2009.98. Epub 2009 Sep 17.

33.

Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells.

Zou J, Maeder ML, Mali P, Pruett-Miller SM, Thibodeau-Beganny S, Chou BK, Chen G, Ye Z, Park IH, Daley GQ, Porteus MH, Joung JK, Cheng L.

Cell Stem Cell. 2009 Jul 2;5(1):97-110. doi: 10.1016/j.stem.2009.05.023. Epub 2009 Jun 18.

34.

High-frequency modification of plant genes using engineered zinc-finger nucleases.

Townsend JA, Wright DA, Winfrey RJ, Fu F, Maeder ML, Joung JK, Voytas DF.

Nature. 2009 May 21;459(7245):442-5. doi: 10.1038/nature07845. Epub 2009 Apr 29.

35.

Differential expression of the Enhancer of split genes in the developing Drosophila midgut.

Maeder ML, Megley C, Eastman DA.

Hereditas. 2009 Feb;146(1):11-8. doi: 10.1111/j.1601-5223.2008.02094.x.

36.

Rapid mutation of endogenous zebrafish genes using zinc finger nucleases made by Oligomerized Pool ENgineering (OPEN).

Foley JE, Yeh JR, Maeder ML, Reyon D, Sander JD, Peterson RT, Joung JK.

PLoS One. 2009;4(2):e4348. doi: 10.1371/journal.pone.0004348. Epub 2009 Feb 9.

37.

Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification.

Maeder ML, Thibodeau-Beganny S, Osiak A, Wright DA, Anthony RM, Eichtinger M, Jiang T, Foley JE, Winfrey RJ, Townsend JA, Unger-Wallace E, Sander JD, Müller-Lerch F, Fu F, Pearlberg J, Göbel C, Dassie JP, Pruett-Miller SM, Porteus MH, Sgroi DC, Iafrate AJ, Dobbs D, McCray PB Jr, Cathomen T, Voytas DF, Joung JK.

Mol Cell. 2008 Jul 25;31(2):294-301. doi: 10.1016/j.molcel.2008.06.016.

38.

Comparison of zinc finger nucleases for use in gene targeting in mammalian cells.

Pruett-Miller SM, Connelly JP, Maeder ML, Joung JK, Porteus MH.

Mol Ther. 2008 Apr;16(4):707-17. doi: 10.1038/mt.2008.20. Epub 2008 Mar 4.

39.

Phylogenetic footprinting analysis in the upstream regulatory regions of the Drosophila enhancer of split genes.

Maeder ML, Polansky BJ, Robson BE, Eastman DA.

Genetics. 2007 Nov;177(3):1377-94.

40.

Discovery of functional elements in 12 Drosophila genomes using evolutionary signatures.

Stark A, Lin MF, Kheradpour P, Pedersen JS, Parts L, Carlson JW, Crosby MA, Rasmussen MD, Roy S, Deoras AN, Ruby JG, Brennecke J; Harvard FlyBase curators; Berkeley Drosophila Genome Project, Hodges E, Hinrichs AS, Caspi A, Paten B, Park SW, Han MV, Maeder ML, Polansky BJ, Robson BE, Aerts S, van Helden J, Hassan B, Gilbert DG, Eastman DA, Rice M, Weir M, Hahn MW, Park Y, Dewey CN, Pachter L, Kent WJ, Haussler D, Lai EC, Bartel DP, Hannon GJ, Kaufman TC, Eisen MB, Clark AG, Smith D, Celniker SE, Gelbart WM, Kellis M.

Nature. 2007 Nov 8;450(7167):219-32.

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