Publications

The following list of publications, authored by Sangamo scientists, collaborators and others, contains original research articles and review articles that are relevant to our ZFP technology and its many applications.


Reviews

Genome editing with engineered zinc finger nucleases.
Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD.

Nat Rev Genet. 2010 Sep;11(9):636-46. Review.PubMed PMID: 20717154.

The discovery of zinc fingers and their applications in gene regulation and genome manipulation.
Klug A.

Annu Rev Biochem. 2010;79:213-31. Review. PubMed PMID: 20192761.

Drug discovery with engineered zinc-finger proteins.
Jamieson AC, Miller JC, Pabo CO.

Nat Rev Drug Discov. 2003 May;2(5):361-8. Review. PubMed PMID:12750739.

Design and selection of novel Cys2His2 zinc finger proteins
Pabo, C. O., Peisach, E., and Grant, R. A.

Annu Rev Biochem 70, 313-40. (2001)


Therapeutic Applications of our ZFN Technology

The following publications relate to the development of ZFP Therapeutics using ZFNs:

Targeted gene therapy and cell reprogramming in Fanconi anemia.
Rio P, Baños R, Lombardo A, Quintana-Bustamante O, Alvarez L, Garate Z, Genovese P, Almarza E, Valeri A, Díez B, Navarro S, Torres Y, Trujillo JP, Murillas R, Segovia JC, Samper E, Surralles J, Gregory PD, Holmes MC, Naldini L, Bueren JA.

EMBO Mol Med. 2014 May 23;6(6):835-48. PubMed PMID: 24859981

Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV.
Tebas P, Stein D, Tang WW, Frank I, Wang SQ, Lee G, Spratt SK, Surosky RT, Giedlin MA, Nichol G, Holmes MC, Gregory PD, Ando DG, Kalos M, Collman RG, Binder-Scholl G, Plesa G, Hwang WT, Levine BL, June CH.

N Engl J Med. 2014 Mar 6;370(10):901-10. PubMed PMID: 24597865

LRRK2 mutations cause mitochondrial DNA damage in iPSC-derived neural cells from Parkinson's disease patients: reversal by gene correction.
Sanders LH, Laganière J, Cooper O, Mak SK, Vu BJ, Huang YA, Paschon DE, Vangipuram M, Sundararajan R, Urnov FD, Langston JW, Gregory PD, Zhang HS, Greenamyre JT, Isacson O, Schüle B.

Neurobiol Dis. 2014 Feb;62:381-6. Epub 2013 Oct 19. PubMed PMID: 24148854

Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection.
Didigu CA, Wilen CB, Wang J, Duong J, Secreto AJ, Danet-Desnoyers GA, Riley JL, Gregory PD, June CH, Holmes MC, Doms RW.

Blood. 2014 Jan 2;123(1):61-9. Epub 2013 Oct 25. PubMed PMID: 24162716

Robust ZFN-mediated genome editing in adult hemophilic mice.
Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA.

Blood. 2013 Nov 7;122(19):3283-7. Epub 2013 Oct 1. PubMed PMID: 24085764

Translating dosage compensation to trisomy 21.
Jiang J, Jing Y, Cost GJ, Chiang JC, Kolpa HJ, Cotton AM, Carone DM, Carone BR, Shivak DA, Guschin DY, Pearl JR, Rebar EJ, Byron M, Gregory PD, Brown CJ, Urnov FD, Hall LL, Lawrence JB.

Nature. 2013 Aug 15;500(7462):296-300. Epub 2013 Jul 17. PubMed PMID: 23863942

Genomic editing of the HIV-1 coreceptor CCR5 in adult hematopoietic stem and progenitor cells using zinc finger nucleases.
Li L, Krymskaya L, Wang J, Henley J, Rao A, Cao LF, Tran CA, Torres-Coronado M, Gardner A, Gonzalez N, Kim K, Liu PQ, Hofer U, Lopez E, Gregory PD, Liu Q, Holmes MC, Cannon PM, Zaia JA, DiGiusto DL.

Mol Ther. 2013 Jun;21(6):1259-69. Epub 2013 Apr 16. PubMed PMID: 23587921

Use of zinc-finger nucleases to knock out the WAS gene in K562 cells: a human cellular model for Wiskott-Aldrich syndrome.
Toscano MG, Anderson P, Muñoz P, Lucena G, Cobo M, Benabdellah K, Gregory PD, Holmes MC, Martin F.

Dis Model Mech. 2013 Mar;6(2):544-54. Epub 2013 Jan 11. PubMed PMID: 23324327

Efficient clinical scale gene modification via zinc finger nuclease-targeted disruption of the HIV co-receptor CCR5.
Maier DA, Brennan AL, Jiang S, Binder-Scholl GK, Lee G, Plesa G, Zheng Z, Cotte J, Carpenito C, Wood T, Spratt SK, Ando D, Gregory P, Holmes MC, Perez EE, Riley JL, Carroll RG, June CH, Levine BL.

Hum Gene Ther. 2013 Mar;24(3):245-58. Epub 2013 Mar 6. PubMed PMID: 23360514

Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation.
Benabdallah BF, Duval A, Rousseau J, Chapdelaine P, Holmes MC, Haddad E, Tremblay JP, Beauséjour CM.

Mol Ther Nucleic Acids. 2013 Jan 29;2:e68. PubMed PMID: 23360951

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR.
Torikai H, Reik A, Liu PQ, Zhou Y, Zhang L, Maiti S, Huls H, Miller JC, Kebriaei P, Rabinovitch B, Lee DA, Champlin RE, Bonini C, Naldini L, Rebar EJ, Gregory PD, Holmes MC, Cooper LJ.

Blood. 2012 Jun 14;119(24):5697-705. Epub 2012 Apr 24. PubMed PMID: 22535661

Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer.
Provasi E, Genovese P, Lombardo A, Magnani Z, Liu PQ, Reik A, Chu V, Paschon DE, Zhang L, Kuball J, Camisa B, Bondanza A, Casorati G, Ponzoni M, Ciceri F, Bordignon C, Greenberg PD, Holmes MC, Gregory PD, Naldini L, Bonini C.

Nat Med. 2012 May;18(5):807-15. PubMed PMID: 22466705

Zinc-finger nuclease editing of human cxcr4 promotes HIV-1 CD4(+) T cell resistance and enrichment.
Yuan J, Wang J, Crain K, Fearns C, Kim KA, Hua KL, Gregory PD, Holmes MC, Torbett BE.

Mol Ther. 2012 Apr;20(4):849-59. Epub 2012 Jan 24. PubMed PMID: 22273578

Engineering HIV-resistant human CD4+ T cells with CXCR4-specific zinc-finger nucleases.
Wilen CB, Wang J, Tilton JC, Miller JC, Kim KA, Rebar EJ, Sherrill-Mix SA, Patro SC, Secreto AJ, Jordan AP, Lee G, Kahn J, Aye PP, Bunnell BA, Lackner AA, Hoxie JA, Danet-Desnoyers GA, Bushman FD, Riley JL, Gregory PD, June CH, Holmes MC, Doms RW.

PLoS Pathog. 2011 Apr;7(4):e1002020. Epub 2011 Apr 14. PubMed PMID: 21533216

Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo.
Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V, Crooks GM, Kohn DB, Gregory PD, Holmes MC, Cannon PM.

Nat Biotechnol. 2010 Aug;28(8):839-47. Epub 2010 Jul 2. PubMed PMID: 20601939.

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases.
Perez EE, Wang J, Miller JC, Jouvenot Y, Kim KA, Liu O, Wang N, Lee G, Bartsevich VV, Lee YL, Guschin DY, Rupniewski I, Waite AJ, Carpenito C, Carroll RG, Orange JS, Urnov FD, Rebar EJ, Ando D, Gregory PD, Riley JL, Holmes MC, June CH.

Nat Biotechnol. 2008 Jul;26(7):808-16. Epub 2008 Jun 29. PubMed PMID: 18587387.

Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases.
Moehle, E A; Rock, J M; Lee, Y L Jouvenot, Y; Dekelver, R C; Gregory, P D ; Urnov, F D; Holmes, M C.

Proc Natl Acad Sci U S A. 2007 Feb 27;104(9):3055-60. Epub 2007 Feb 20. PubMed PMID: 17360608; PubMed Central PMCID: PMC1802009.

Highly efficient endogenous human gene correction using designed zinc-finger nucleases.
Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S, Jamieson AC, Porteus MH, Gregory PD, Holmes MC.

Nature. 2005 Jun2;435(7042):646-51. Epub 2005 Apr 3. PubMed PMID: 15806097.


Therapeutic Applications of our ZFP TF Technology

The following publications relate to the development of therapeutics based on ZFP TFs:

Reactivation of developmentally silenced globin genes by forced chromatin looping.
Deng W, Rupon JW, Krivega I, Breda L, Motta I, Jahn KS, Reik A, Gregory PD, Rivella S, Dean A, Blobel GA.

Cell. 2014 Aug 14;158(4):849-60. PubMed PMID: 25126789

A designed zinc-finger transcriptional repressor of phospholamban improves function of the failing heart.
Zhang HS, Liu D, Huang Y, Schmidt S, Hickey R, Guschin D, Su H, Jovin IS, Kunis M, Hinkley S, Liang Y, Hinh L, Spratt SK, Case CC, Rebar EJ, Ehrlich BE, Gregory PD, Giordano FJ.

Mol Ther. 2012 Aug;20(8):1508-15. Epub 2012 Jul 24. Erratum in: Mol Ther. 2012 Dec;20(12):2370. Ehrlich, Barbara [corrected to Ehrlich, Barbara E]. PubMed PMID: 22828502

An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease.
Laganiere J, Kells AP, Lai JT, Guschin D, Paschon DE, Meng X, Fong LK, Yu Q, Rebar EJ, Gregory PD, Bankiewicz KS, Forsayeth J, Zhang HS.

J Neurosci. 2010 Dec 8;30(49):16469-74. PubMed PMID: 21147986.

Engineered zinc finger protein-mediated VEGF-a activation restores deficient VEGF-A in sensory neurons in experimental diabetes.
Pawson EJ, Duran-Jimenez B, Surosky R, Brooke HE, Spratt SK, Tomlinson DR,
Gardiner NJ. 

Diabetes. 2010 Feb;59(2):509-18. Epub 2009 Nov 23. PubMed PMID: 19934008; PubMed Central PMCID: PMC2809974.

An Engineered Transcription Factor Which Activates VEGF-A Enhances Recovery After
Spinal Cord Injury.

Liu Y, Figley S, Spratt SK, Lee G, Ando D, Surosky R, Fehlings MG. 

Neurobiol Dis. 2010 Feb;37(2):384-93. Epub 2009 Oct 29. PubMed PMID: 19879362.

Gene transfer of an engineered zinc finger protein enhances the anti-angiogenic defense system.
Yokoi K, Zhang HS, Kachi S, Balaggan KS, Yu Q, Guschin D, Kunis M, Surosky R,Africa LM, Bainbridge JW, Spratt SK, Gregory PD, Ali RR, Campochiaro PA.

Mol Ther. 2007 Nov;15(11):1917-23. Epub 2007 Aug 14. PubMed PMID:17700545.

Regulation of endogenous gene expression using small molecule-controlled engineered zinc-finger protein transcription factors.
Dent CL, Lau G, Drake EA, Yoon A, Case CC, Gregory PD.

Gene Ther. 2007 Sep;14(18):1362-9. Epub 2007 Jul 19. PubMed PMID: 17637799.

Ocular gene transfer with self-complementary AAV vectors.
Yokoi K, Kachi S, Zhang HS, Gregory PD, Spratt SK, Samulski RJ, Campochiaro PA.

Invest Ophthalmol Vis Sci. 2007 Jul;48(7):3324-8. PubMed PMID: 17591905.

Gene transfer of an engineered transcription factor promoting expression of VEGF-A protects against experimental diabetic neuropathy.
Price SA, Dent C, Duran-Jimenez B, Liang Y, Zhang L, Rebar EJ, Case CC, Gregory PD, Martin TJ, Spratt SK, Tomlinson DR.

Diabetes. 2006 Jun;55(6):1847-54. PubMed PMID: 16731852.

Repression of vascular endothelial growth factor A in glioblastoma cells using engineered zinc finger transcription factors.
Snowden AW, Zhang L, Urnov F, Dent C, Jouvenot Y, Zhong X, Rebar EJ, Jamieson AC, Zhang HS, Tan S, Case CC, Pabo CO, Wolffe AP, Gregory PD.

Cancer Res. 2003 Dec 15;63(24):8968-76. PubMed PMID: 14695215

Zinc-finger protein-targeted gene regulation: genomewide single-gene specificity.
Tan S, Guschin D, Davalos A, Lee YL, Snowden AW, Jouvenot Y, Zhang HS, Howes K, McNamara AR, Lai A, Ullman C, Reynolds L, Moore M, Isalan M, Berg LP, Campos B, Qi H, Spratt SK, Case CC, Pabo CO, Campisi J, Gregory PD.

Proc Natl Acad Sci U S A. 2003 Oct 14;100(21):11997-2002. Epub 2003 Sep 26. PubMed PMID: 14514889; PubMed Central PMCID: PMC218702.

Targeted regulation of imprinted genes by synthetic zinc-finger transcription factors.
Jouvenot Y, Ginjala V, Zhang L, Liu PQ, Oshimura M, Feinberg AP, Wolffe AP, Ohlsson R, Gregory PD.

Gene Ther. 2003 Mar;10(6):513-22. PubMed PMID: 12621455.

Gene-specific targeting of H3K9 methylation is sufficient for initiating repression in vivo.
Snowden AW, Gregory PD, Case CC, Pabo CO.

Curr Biol. 2002 Dec 23;12(24):2159-66. PubMed PMID: 12498693.

PPARgamma knockdown by engineered transcription factors: exogenous PPARgamma2 but not PPARgamma1 reactivates adipogenesis.
Ren, D., Collingwood, T. N., Rebar, E. J., Wolffe, A. P., and Camp, H. S.

Genes Dev 16, 27-32(2002).

Induction of angiogenesis in a mouse model using engineered transcription factors
Rebar, E. J., Huang, Y., Hickey, R., Nath, A. K., Meoli, D., Nath, S., Chen, B., Xu, L., Liang, Y., Jamieson, A. C., et al.

Nat Med 8, 1427-32(2002).


Research Applications of our ZFN and ZFP TF Technology

Stem Cell Applications

ZFNs

Genetic and molecular identification of three human TPP1 functions in telomerase action: recruitment, activation, and homeostasis set point regulation.
Sexton AN, Regalado SG, Lai CS, Cost GJ, O'Neil CM, Urnov FD, Gregory PD, Jaenisch R, Collins K, Hockemeyer D.

Genes Dev. 2014 Sep 1;28(17):1885-99. Epub 2014 Aug 15. PubMed PMID: 25128433.

Targeted genome editing in human repopulating haematopoietic stem cells.
Genovese P, Schiroli G, Escobar G, Di Tomaso T, Firrito C, Calabria A, Moi D, Mazzieri R, Bonini C, Holmes MC, Gregory PD, van der Burg M, Gentner B, Montini E, Lombardo A, Naldini L.

Nature. 2014 Jun 12;510(7504):235-40. Epub 2014 May 28. PubMed PMID: 24870228

Human intestinal tissue with adult stem cell properties derived from pluripotent stem cells.
Forster R, Chiba K, Schaeffer L, Regalado SG, Lai CS, Gao Q, Kiani S, Farin HF, Clevers H, Cost GJ, Chan A, Rebar EJ, Urnov FD, Gregory PD, Pachter L, Jaenisch R, Hockemeyer D.

Stem Cell Reports. 2014 Jun 3;2(6):838-52. eCollection 2014 Jun 3. PubMed PMID: 24936470

Genome editing of human embryonic stem cells and induced pluripotent stem cells with zinc finger nucleases for cellular imaging.
Wang Y, Zhang WY, Hu S, Lan F, Lee AS, Huber B, Lisowski L, Liang P, Huang M, de Almeida PE, Won JH, Sun N, Robbins RC, Kay MA, Urnov FD, Wu JC.

Circ Res. 2012 Dec 7;111(12):1494-503. Epub 2012 Sep 11. PubMed PMID: 22967807

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases.
Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver RC, Katibah GE, Amora R, Boydston EA, Zeitler B, Meng X, Miller JC, Zhang L, Rebar EJ,Gregory PD, Urnov FD, Jaenisch R.

Nat Biotechnol. 2009 Sep;27(9):851-7. Epub 2009 Aug 13. PubMed PMID: 19680244.

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery.
Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, Ando D, Urnov FD, Galli C, Gregory PD, Holmes MC, Naldini L.

Nat Biotechnol. 2007 Nov;25(11):1298-306. Epub 2007 Oct 28. PubMed PMID: 17965707.

ZFP TFs

Engineered zinc finger proteins for controlling stem cell fate.
Bartsevich VV, Miller JC, Case CC, Pabo CO.

Stem Cells. 2003;21(6):632-7. PubMed PMID:14595122.

Creation of Transgenic Animals

Knockout rats via embryo microinjection of zinc-finger nucleases.
Geurts AM, Cost GJ, Freyvert Y, Zeitler B, Miller JC, Choi VM, Jenkins SS, Wood A, Cui X, Meng X, Vincent A, Lam S, Michalkiewicz M, Schilling R, Foeckler J, Kalloway S, Weiler H, Ménoret S, Anegon I, Davis GD, Zhang L, Rebar EJ,Gregory PD, Urnov FD, Jacob HJ, Buelow R.

Science. 2009 Jul 24;325(5939):433. PubMed PMID:19628861; PubMed Central PMCID: PMC2831805.

Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases.
Doyon Y, McCammon JM, Miller JC, Faraji F, Ngo C, Katibah GE, Amora R, Hocking TD, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Amacher SL.

Nat Biotechnol. 2008 Jun;26(6):702-8. Epub 2008 May 25. PubMed PMID: 18500334; PubMed Central PMCID: PMC2674762.

Use of ZFNs and ZFP TFs in Plants

ZFNs

Trait stacking via targeted genome editing.
Ainley WM, Sastry-Dent L, Welter ME, Murray MG, Zeitler B, Amora R, Corbin DR, Miles RR, Arnold NL, Strange TL, Simpson MA, Cao Z, Carroll C, Pawelczak KS, Blue R, West K, Rowland LM, Perkins D, Samuel P, Dewes CM, Shen L, Sriram S, Evans SL, Rebar EJ, Zhang L, Gregory PD, Urnov FD, Webb SR, Petolino JF.

Plant Biotechnol J. 2013 Dec;11(9):1126-34. Epub 2013 Aug 19. PubMed PMID: 23953646

Targeted transgene integration in plant cells using designed zinc finger nucleases.
Cai CQ, Doyon Y, Ainley WM, Miller JC, Dekelver RC, Moehle EA, Rock JM, LeeYL, Garrison R, Schulenberg L, Blue R, Worden A, Baker L, Faraji F, Zhang L, Holmes MC, Rebar EJ, Collingwood TN, Rubin-Wilson B, Gregory PD, Urnov FD, Petolino JF.

Plant Mol Biol. 2009 Apr;69(6):699-709. Epub 2008 Dec 27.PubMed PMID: 19112554.

Precise genome modification in the crop species Zea mays using zinc-finger nucleases.
Shukla VK, Doyon Y, Miller JC, DeKelver RC, Moehle EA, Worden SE, Mitchell JC, Arnold NL, Gopalan S, Meng X, Choi VM, Rock JM, Wu YY, Katibah GE, Zhifang G, McCaskill D, Simpson MA, Blakeslee B, Greenwalt SA, Butler HJ, Hinkley SJ, Zhang L, Rebar EJ, Gregory PD, Urnov FD.

Nature. 2009 May21;459(7245):437-41. Epub 2009 Apr 29. PubMed PMID: 19404259.

ZFP TFs

Activation domains for controlling plant gene expression using designed transcription factors.
Li J, Blue R, Zeitler B, Strange TL, Pearl JR, Huizinga DH, Evans S, Gregory PD, Urnov FD, Petolino JF.

Plant Biotechnol J. 2013 Aug;11(6):671-80. Epub 2013 Mar 22. PubMed PMID: 23521778

Transcriptional activation of Brassica napus β-ketoacyl-ACP synthase II with an engineered zinc finger protein transcription factor.
Gupta M, DeKelver RC, Palta A, Clifford C, Gopalan S, Miller JC, Novak S, Desloover D, Gachotte D, Connell J, Flook J, Patterson T, Robbins K, Rebar EJ, Gregory PD, Urnov FD, Petolino JF.

Plant Biotechnol J. 2012 Sep;10(7):783-91. Epub 2012 Apr 23. PubMed PMID: 22520333

Gene regulation in planta by plant-derived engineered zinc finger protein transcription factors.
Holmes-Davis R, Li G, Jamieson AC, Rebar EJ, Liu Q, Kong Y, Case CC, Gregory PD.

Plant Mol Biol. 2005 Feb;57(3):411-23. PubMed PMID:15830130.

Cell Biology Applications

Drug resistance. K13-propeller mutations confer artemisinin resistance in Plasmodium falciparum clinical isolates.
Straimer J, Gnädig NF, Witkowski B, Amaratunga C, Duru V, Ramadani AP, Dacheux M, Khim N, Zhang L, Lam S, Gregory PD, Urnov FD, Mercereau-Puijalon O, Benoit-Vical F, Fairhurst RM, Ménard D, Fidock DA.

Science. 2015 Jan 23;347(6220):428-31. Epub 2014 Dec 11. PubMed PMID: 25502314

Cancer translocations in human cells induced by zinc finger and TALE nucleases.
Piganeau M, Ghezraoui H, De Cian A, Guittat L, Tomishima M, Perrouault L, René O, Katibah GE, Zhang L, Holmes MC, Doyon Y, Concordet JP, Giovannangeli C, Jasin M, Brunet E.

Genome Res. 2013 Jul;23(7):1182-93. Epub 2013 Apr 8. PubMed PMID: 23568838

Controlling long-range genomic interactions at a native locus by targeted tethering of a looping factor.
Deng W, Lee J, Wang H, Miller J, Reik A, Gregory PD, Dean A, Blobel GA.

Cell. 2012 Jun 8;149(6):1233-44. PubMed PMID: 22682246

Rapid and efficient clathrin-mediated endocytosis revealed in genome-edited mammalian cells
Doyon JB, Zeitler B, Cheng J, Cheng AT, Cherone JM, Santiago Y, Lee AH, Vo TD, Doyon Y, Miller JC, Paschon DE, Zhang L, Rebar EJ, Gregory PD, Urnov FD, Drubin DG.

Nat Cell Biol. 2011 Feb 6. [Epub ahead of print] PubMed PMID:21297641.

Distinct factors control histone variant H3.3 localization at specific genomic regions.
Goldberg AD, Banaszynski LA, Noh KM, Lewis PW, Elsaesser SJ, Stadler S, Dewell S, Law M, Guo X, Li X, Wen D, Chapgier A, DeKelver RC, Miller JC, Lee YL, Boydston EA, Holmes MC, Gregory PD, Greally JM, Rafii S, Yang C, Scambler PJ, Garrick D, Gibbons RJ, Higgs DR, Cristea IM, Urnov FD, Zheng D, Allis CD.

Cell. 2010 Mar 5;140(5):678-91. PubMed PMID: 20211137; PubMed Central PMCID: PMC2885838.

Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases.
Santiago Y, Chan E, Liu PQ, Orlando S, Zhang L, Urnov FD, Holmes MC, Guschin D, Waite A, Miller JC, Rebar EJ, Gregory PD, Klug A, Collingwood TN.

Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5809-14. Epub 2008 Mar 21. PubMed PMID: 18359850; PubMed Central PMCID: PMC2299223.

Histone deimination antagonizes arginine methylation.
Cuthbert GL, Daujat S, Snowden AW, Erdjument-Bromage H, Hagiwara T, Yamada M, Schneider R, Gregory PD, Tempst P, Bannister AJ, Kouzarides T.

Cell. 2004 Sep 3;118(5):545-53.PubMed PMID: 15339660.

Use of ZFP TFs in Drosophila

Controlling gene expression in Drosophila using engineered zinc finger proteintranscription factors.
Jamieson AC, Guan B, Cradick TJ, Xiao H, Holmes MC, Gregory PD, Carroll PM.

Biochem Biophys Res Commun. 2006 Sep 29;348(3):873-9. Epub 2006 Jul 31. PubMed PMID: 16899226.

Creation of Novel Cell Lines for More Efficient Protein Production

Highly efficient deletion of FUT8 in CHO cell lines using zinc-finger nucleases yields cells that produce completely nonfucosylated antibodies.
Malphettes L, Freyvert Y, Chang J, Liu PQ, Chan E, Miller JC, Zhou Z, Nguyen T, Tsai C, Snowden AW, Collingwood TN, Gregory PD, Cost GJ.

Biotechnol Bioeng. 2010 Aug 1;106(5):774-83. PubMed PMID: 20564614.

Generation of a triple-gene knockout mammalian cell line using engineered zinc-finger nucleases.
Liu PQ, Chan EM, Cost GJ, Zhang L, Wang J, Miller JC, Guschin DY, Reik A, Holmes MC, Mott JE, Collingwood TN, Gregory PD.

Biotechnol Bioeng. 2010 May 1;106(1):97-105. PubMed PMID: 20047187.

BAK and BAX deletion using zinc-finger nucleases yields apoptosis-resistant CHO cells.
Cost GJ, Freyvert Y, Vafiadis A, Santiago Y, Miller JC, Rebar E, Collingwood TN, Snowden A, Gregory PD.

Biotechnol Bioeng. 2010 Feb 1;105(2):330-40. PubMed PMID: 19777580.

ZFP TF’s for Enhanced Protein Production

Enhanced protein production by engineered zinc finger proteins.
Reik A, Zhou Y, Collingwood TN, Warfe L, Bartsevich V, Kong Y, Henning KA, Fallentine BK, Zhang L, Zhong X, Jouvenot Y, Jamieson AC, Rebar EJ, Case CC, Korman A, Li XY, Black A, King DJ, Gregory PD

Biotechnol Bioeng. 2007 Aug 1;97(5):1180-9.PubMed PMID: 17171718.

Isogenic human cell lines for drug discovery: regulation of target gene expression by engineered zinc-finger protein transcription factors.
Liu PQ, Tan S, Mendel MC, Murrills RJ, Bhat BM, Schlag B, Samuel R, Matteo JJ, de la Rosa R, Howes K, Reik A, Case CC, Bex FJ, Young K, Gregory PD.

J Biomol Screen. 2005 Jun;10(4):304-13. PubMed PMID: 15964931.

ZFN Gene-Editing Topics

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors.
Torikai H, Reik A, Soldner F, Warren EH, Yuen C, Zhou Y, Crossland DL, Huls H, Littman N, Zhang Z, Tykodi SS, Kebriaei P, Lee DA, Miller JC, Rebar EJ, Holmes MC, Jaenisch R, Champlin RE, Gregory PD, Cooper LJ.

Blood. 2013 Aug 22;122(8):1341-9. Epub 2013 Jun 5. PubMed PMID: 23741009

In vivo cleavage of transgene donors promotes nuclease-mediated targeted integration.
Cristea S, Freyvert Y, Santiago Y, Holmes MC, Urnov FD, Gregory PD, Cost GJ.

Biotechnol Bioeng. 2013 Mar;110(3):871-80. Epub 2012 Oct 23. PubMed PMID: 23042119

Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme.
Wang J, Friedman G, Doyon Y, Wang NS, Li CJ, Miller JC, Hua KL, Yan JJ, Babiarz JE, Gregory PD, Holmes MC.

Genome Res. 2012 Jul;22(7):1316-26. Epub 2012 Mar 20. PubMed PMID: 22434427

Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology.
Orlando SJ, Santiago Y, DeKelver RC, Freyvert Y, Boydston EA, Moehle EA, Choi VM, Gopalan SM, Lou JF, Li J, Miller JC, Holmes MC, Gregory PD, Urnov FD, Cost GJ.

Nucleic Acids Res. 2010 Aug;38(15):e152. Epub 2010 Jun 8. PubMed PMID: 20530528; PubMed Central PMCID:PMC2926620.

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome.
DeKelver RC, Choi VM, Moehle EA, Paschon DE, Hockemeyer D, Meijsing SH, Sancak Y, Cui X, Steine EJ, Miller JC, Tam P, Bartsevich VV, Meng X, Rupniewski I,Gopalan SM, Sun HC, Pitz KJ, Rock JM, Zhang L, Davis GD, Rebar EJ, Cheeseman IM, Yamamoto KR, Sabatini DM, Jaenisch R, Gregory PD, Urnov FD.

Genome Res. 2010 Aug;20(8):1133-42. Epub 2010 May 27. PubMed PMID: 20508142; PubMed
Central PMCID: PMC2909576.

Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform.
Benabdallah BF, Allard E, Yao S, Friedman G, Gregory PD, Eliopoulos N, Fradette J, Spees JL, Haddad E, Holmes MC, Beauséjour CM.

Cytotherapy. 2010 May;12(3):394-9. PubMed PMID: 20331411.

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