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Items: 1 to 50 of 100

1.

Inhibition of replication of hepatitis B virus using transcriptional repressors that target the viral DNA.

Bloom K, Kaldine H, Cathomen T, Mussolino C, Ely A, Arbuthnot P.

BMC Infect Dis. 2019 Sep 12;19(1):802. doi: 10.1186/s12879-019-4436-y.

2.

Retroviral UNC13D Gene Transfer Restores Cytotoxic Activity of T Cells Derived from Familial Hemophagocytic Lymphohistiocytosis Type 3 Patients In Vitro.

Dettmer V, Bloom K, Gross M, Weissert K, Aichele P, Ehl S, Cathomen T.

Hum Gene Ther. 2019 Aug;30(8):975-984. doi: 10.1089/hum.2019.025. Epub 2019 May 21.

PMID:
31032638
3.

Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.

Patsali P, Turchiano G, Papasavva P, Romito M, Loucari CC, Stephanou C, Christou S, Sitarou M, Mussolino C, Cornu TI, Antoniou MN, Lederer CW, Cathomen T, Kleanthous M.

Haematologica. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19. No abstract available.

4.

The Human Genome Editing Race: Loosening Regulatory Standards for Commercial Advantage?

Cathomen T, Schüle S, Schüßler-Lenz M, Abou-El-Enein M.

Trends Biotechnol. 2019 Feb;37(2):120-123. doi: 10.1016/j.tibtech.2018.06.005. Epub 2018 Jul 13. Review.

PMID:
30017092
5.

Designer epigenome modifiers enable robust and sustained gene silencing in clinically relevant human cells.

Mlambo T, Nitsch S, Hildenbeutel M, Romito M, Müller M, Bossen C, Diederichs S, Cornu TI, Cathomen T, Mussolino C.

Nucleic Acids Res. 2018 May 18;46(9):4456-4468. doi: 10.1093/nar/gky171.

6.

Homologous recombination mediates stable Fah gene integration and phenotypic correction in tyrosinaemia mouse-model.

Junge N, Yuan Q, Vu TH, Krooss S, Bednarski C, Balakrishnan A, Cathomen T, Manns MP, Baumann U, Sharma AD, Ott M.

World J Hepatol. 2018 Feb 27;10(2):277-286. doi: 10.4254/wjh.v10.i2.277.

7.

Efficient Recombinase-Mediated Cassette Exchange in hPSCs to Study the Hepatocyte Lineage Reveals AAVS1 Locus-Mediated Transgene Inhibition.

Ordovás L, Boon R, Pistoni M, Chen Y, Wolfs E, Guo W, Sambathkumar R, Bobis-Wozowicz S, Helsen N, Vanhove J, Berckmans P, Cai Q, Vanuytsel K, Eggermont K, Vanslembrouck V, Schmidt BZ, Raitano S, Van Den Bosch L, Nahmias Y, Cathomen T, Struys T, Verfaillie CM.

Stem Cell Reports. 2018 Feb 13;10(2):673. doi: 10.1016/j.stemcr.2018.01.034. No abstract available.

8.

TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis.

Kuhn A, Ackermann M, Mussolino C, Cathomen T, Lachmann N, Moritz T.

Sci Rep. 2017 Nov 9;7(1):15195. doi: 10.1038/s41598-017-14566-8.

9.

Human Genome Editing in the Clinic: New Challenges in Regulatory Benefit-Risk Assessment.

Abou-El-Enein M, Cathomen T, Ivics Z, June CH, Renner M, Schneider CK, Bauer G.

Cell Stem Cell. 2017 Oct 5;21(4):427-430. doi: 10.1016/j.stem.2017.09.007.

10.

Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.

Alzubi J, Pallant C, Mussolino C, Howe SJ, Thrasher AJ, Cathomen T.

Sci Rep. 2017 Sep 29;7(1):12475. doi: 10.1038/s41598-017-12750-4.

11.

Generation of a gene-corrected isogenic control iPSC line from cystic fibrosis patient-specific iPSCs homozygous for p.Phe508del mutation mediated by TALENs and ssODN.

Merkert S, Bednarski C, Göhring G, Cathomen T, Martin U.

Stem Cell Res. 2017 Aug;23:95-97. doi: 10.1016/j.scr.2017.07.010. Epub 2017 Jul 11.

12.

Genome and Epigenome Editing to Treat Disorders of the Hematopoietic System.

Mussolino C, Alzubi J, Pennucci V, Turchiano G, Cathomen T.

Hum Gene Ther. 2017 Nov;28(11):1105-1115. doi: 10.1089/hum.2017.149. Epub 2017 Aug 14. Review.

PMID:
28806883
13.

Traceless Targeting and Isolation of Gene-Edited Immortalized Keratinocytes from Epidermolysis Bullosa Simplex Patients.

Aushev M, Koller U, Mussolino C, Cathomen T, Reichelt J.

Mol Ther Methods Clin Dev. 2017 Jul 5;6:112-123. doi: 10.1016/j.omtm.2017.06.008. eCollection 2017 Sep 15.

14.

Refining strategies to translate genome editing to the clinic.

Cornu TI, Mussolino C, Cathomen T.

Nat Med. 2017 Apr 3;23(4):415-423. doi: 10.1038/nm.4313. Review.

PMID:
28388605
15.

Therapeutic genome editing with engineered nucleases.

Haas SA, Dettmer V, Cathomen T.

Hamostaseologie. 2017 Jan 31;37(1):45-52. doi: 10.5482/HAMO-16-09-0035. Epub 2017 Jan 10. Review.

PMID:
28070592
16.

Improved bi-allelic modification of a transcriptionally silent locus in patient-derived iPSC by Cas9 nickase.

Eggenschwiler R, Moslem M, Fráguas MS, Galla M, Papp O, Naujock M, Fonfara I, Gensch I, Wähner A, Beh-Pajooh A, Mussolino C, Tauscher M, Steinemann D, Wegner F, Petri S, Schambach A, Charpentier E, Cathomen T, Cantz T.

Sci Rep. 2016 Dec 2;6:38198. doi: 10.1038/srep38198.

17.

Failure to detect DNA-guided genome editing using Natronobacterium gregoryi Argonaute.

Lee SH, Turchiano G, Ata H, Nowsheen S, Romito M, Lou Z, Ryu SM, Ekker SC, Cathomen T, Kim JS.

Nat Biotechnol. 2016 Nov 28;35(1):17-18. doi: 10.1038/nbt.3753. No abstract available.

18.

Targeted Integration of a Super-Exon into the CFTR Locus Leads to Functional Correction of a Cystic Fibrosis Cell Line Model.

Bednarski C, Tomczak K, Vom Hövel B, Weber WM, Cathomen T.

PLoS One. 2016 Aug 15;11(8):e0161072. doi: 10.1371/journal.pone.0161072. eCollection 2016.

19.

Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome.

Müller M, Lee CM, Gasiunas G, Davis TH, Cradick TJ, Siksnys V, Bao G, Cathomen T, Mussolino C.

Mol Ther. 2016 Mar;24(3):636-44. doi: 10.1038/mt.2015.218. Epub 2015 Dec 14.

20.

Efficient Recombinase-Mediated Cassette Exchange in hPSCs to Study the Hepatocyte Lineage Reveals AAVS1 Locus-Mediated Transgene Inhibition.

Ordovás L, Boon R, Pistoni M, Chen Y, Wolfs E, Guo W, Sambathkumar R, Bobis-Wozowicz S, Helsen N, Vanhove J, Berckmans P, Cai Q, Vanuytsel K, Eggermont K, Vanslembrouck V, Schmidt BZ, Raitano S, Van Den Bosch L, Nahmias Y, Cathomen T, Struys T, Verfaillie CM.

Stem Cell Reports. 2015 Nov 10;5(5):918-931. doi: 10.1016/j.stemcr.2015.09.004. Epub 2015 Oct 8. Erratum in: Stem Cell Reports. 2018 Feb 13;10 (2):673.

21.

Proven and novel strategies for efficient editing of the human genome.

Mussolino C, Mlambo T, Cathomen T.

Curr Opin Pharmacol. 2015 Oct;24:105-12. doi: 10.1016/j.coph.2015.08.008. Epub 2015 Sep 4. Review.

PMID:
26342909
22.

TALEN-mediated functional correction of X-linked chronic granulomatous disease in patient-derived induced pluripotent stem cells.

Dreyer AK, Hoffmann D, Lachmann N, Ackermann M, Steinemann D, Timm B, Siler U, Reichenbach J, Grez M, Moritz T, Schambach A, Cathomen T.

Biomaterials. 2015 Nov;69:191-200. doi: 10.1016/j.biomaterials.2015.07.057. Epub 2015 Aug 3.

PMID:
26295532
23.

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.

24.

Genome editing technologies: defining a path to clinic.

Corrigan-Curay J, O'Reilly M, Kohn DB, Cannon PM, Bao G, Bushman FD, Carroll D, Cathomen T, Joung JK, Roth D, Sadelain M, Scharenberg AM, von Kalle C, Zhang F, Jambou R, Rosenthal E, Hassani M, Singh A, Porteus MH.

Mol Ther. 2015 May;23(5):796-806. doi: 10.1038/mt.2015.54. No abstract available.

25.

Editing CCR5: a novel approach to HIV gene therapy.

Cornu TI, Mussolino C, Bloom K, Cathomen T.

Adv Exp Med Biol. 2015;848:117-30. doi: 10.1007/978-1-4939-2432-5_6. Review.

PMID:
25757618
26.

Deciphering the impact of parameters influencing transgene expression kinetics after repeated cell transduction with integration-deficient retroviral vectors.

Schott JW, Jaeschke NM, Hoffmann D, Maetzig T, Ballmaier M, Godinho T, Cathomen T, Schambach A.

Cytometry A. 2015 May;87(5):405-18. doi: 10.1002/cyto.a.22650. Epub 2015 Feb 27.

27.

Restoration of progranulin expression rescues cortical neuron generation in an induced pluripotent stem cell model of frontotemporal dementia.

Raitano S, Ordovàs L, De Muynck L, Guo W, Espuny-Camacho I, Geraerts M, Khurana S, Vanuytsel K, Tóth BI, Voets T, Vandenberghe R, Cathomen T, Van Den Bosch L, Vanderhaeghen P, Van Damme P, Verfaillie CM.

Stem Cell Reports. 2015 Jan 13;4(1):16-24. doi: 10.1016/j.stemcr.2014.12.001. Epub 2014 Dec 31.

28.

Autosomal dominant immune dysregulation syndrome in humans with CTLA4 mutations.

Schubert D, Bode C, Kenefeck R, Hou TZ, Wing JB, Kennedy A, Bulashevska A, Petersen BS, Schäffer AA, Grüning BA, Unger S, Frede N, Baumann U, Witte T, Schmidt RE, Dueckers G, Niehues T, Seneviratne S, Kanariou M, Speckmann C, Ehl S, Rensing-Ehl A, Warnatz K, Rakhmanov M, Thimme R, Hasselblatt P, Emmerich F, Cathomen T, Backofen R, Fisch P, Seidl M, May A, Schmitt-Graeff A, Ikemizu S, Salzer U, Franke A, Sakaguchi S, Walker LSK, Sansom DM, Grimbacher B.

Nat Med. 2014 Dec;20(12):1410-1416. doi: 10.1038/nm.3746. Epub 2014 Oct 20.

29.

Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases.

Mock U, Riecken K, Berdien B, Qasim W, Chan E, Cathomen T, Fehse B.

Sci Rep. 2014 Sep 18;4:6409. doi: 10.1038/srep06409.

30.

Adenoviral vector DNA for accurate genome editing with engineered nucleases.

Holkers M, Maggio I, Henriques SF, Janssen JM, Cathomen T, Gonçalves MA.

Nat Methods. 2014 Oct;11(10):1051-7. doi: 10.1038/nmeth.3075. Epub 2014 Aug 24.

PMID:
25152084
31.

Translating the genomic revolution - targeted genome editing in primates.

Cathomen T, Ehl S.

N Engl J Med. 2014 Jun 12;370(24):2342-5. doi: 10.1056/NEJMcibr1403629. No abstract available.

PMID:
24918378
32.

Transfusion-transmitted hepatitis E in Germany, 2013.

Huzly D, Umhau M, Bettinger D, Cathomen T, Emmerich F, Hasselblatt P, Hengel H, Herzog R, Kappert O, Maassen S, Schorb E, Schulz-Huotari C, Thimme R, Unmüssig R, Wenzel JJ, Panning M.

Euro Surveill. 2014 May 29;19(21). pii: 20812.

33.

TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity.

Mussolino C, Alzubi J, Fine EJ, Morbitzer R, Cradick TJ, Lahaye T, Bao G, Cathomen T.

Nucleic Acids Res. 2014 Jun;42(10):6762-73. doi: 10.1093/nar/gku305. Epub 2014 May 3.

34.

Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery.

Bobis-Wozowicz S, Galla M, Alzubi J, Kuehle J, Baum C, Schambach A, Cathomen T.

Sci Rep. 2014 Apr 11;4:4656. doi: 10.1038/srep04656.

35.

Efficient designer nuclease-based homologous recombination enables direct PCR screening for footprintless targeted human pluripotent stem cells.

Merkert S, Wunderlich S, Bednarski C, Beier J, Haase A, Dreyer AK, Schwanke K, Meyer J, Göhring G, Cathomen T, Martin U.

Stem Cell Reports. 2014 Jan 14;2(1):107-18. doi: 10.1016/j.stemcr.2013.12.003. eCollection 2014 Jan 14.

36.

Construction and characterization of adenoviral vectors for the delivery of TALENs into human cells.

Holkers M, Cathomen T, Gonçalves MA.

Methods. 2014 Sep;69(2):179-87. doi: 10.1016/j.ymeth.2014.02.017. Epub 2014 Feb 20.

PMID:
24561826
37.

TALE-PvuII fusion proteins--novel tools for gene targeting.

Yanik M, Alzubi J, Lahaye T, Cathomen T, Pingoud A, Wende W.

PLoS One. 2013 Dec 5;8(12):e82539. doi: 10.1371/journal.pone.0082539. eCollection 2013.

38.

Histone deacetylase inhibition rescues gene knockout levels achieved with integrase-defective lentiviral vectors encoding zinc-finger nucleases.

Pelascini LP, Maggio I, Liu J, Holkers M, Cathomen T, Gonçalves MA.

Hum Gene Ther Methods. 2013 Dec;24(6):399-411. doi: 10.1089/hgtb.2013.107. Epub 2013 Oct 29.

39.

Inactivation of hepatitis B virus replication in cultured cells and in vivo with engineered transcription activator-like effector nucleases.

Bloom K, Ely A, Mussolino C, Cathomen T, Arbuthnot P.

Mol Ther. 2013 Oct;21(10):1889-97. doi: 10.1038/mt.2013.170. Epub 2013 Jul 25.

40.

RNA guides genome engineering.

Mussolino C, Cathomen T.

Nat Biotechnol. 2013 Mar;31(3):208-9. doi: 10.1038/nbt.2527. No abstract available.

PMID:
23471067
41.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G.

Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. No abstract available.

42.

Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells.

Holkers M, Maggio I, Liu J, Janssen JM, Miselli F, Mussolino C, Recchia A, Cathomen T, Gonçalves MA.

Nucleic Acids Res. 2013 Mar 1;41(5):e63. doi: 10.1093/nar/gks1446. Epub 2012 Dec 28.

43.

The nontoxic cell cycle modulator indirubin augments transduction of adeno-associated viral vectors and zinc-finger nuclease-mediated gene targeting.

Rahman SH, Bobis-Wozowicz S, Chatterjee D, Gellhaus K, Pars K, Heilbronn R, Jacobs R, Cathomen T.

Hum Gene Ther. 2013 Jan;24(1):67-77. doi: 10.1089/hum.2012.168. Epub 2012 Dec 10.

44.

MicroRNA-221 overexpression accelerates hepatocyte proliferation during liver regeneration.

Yuan Q, Loya K, Rani B, Möbus S, Balakrishnan A, Lamle J, Cathomen T, Vogel A, Manns MP, Ott M, Cantz T, Sharma AD.

Hepatology. 2013 Jan;57(1):299-310. doi: 10.1002/hep.25984.

PMID:
22821679
45.

Retargeting sleeping beauty transposon insertions by engineered zinc finger DNA-binding domains.

Voigt K, Gogol-Döring A, Miskey C, Chen W, Cathomen T, Izsvák Z, Ivics Z.

Mol Ther. 2012 Oct;20(10):1852-62. doi: 10.1038/mt.2012.126. Epub 2012 Jul 10.

46.

Retargeting transposon insertions by the adeno-associated virus Rep protein.

Ammar I, Gogol-Döring A, Miskey C, Chen W, Cathomen T, Izsvák Z, Ivics Z.

Nucleic Acids Res. 2012 Aug;40(14):6693-712. doi: 10.1093/nar/gks317. Epub 2012 Apr 19.

47.

Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects.

Ramirez CL, Certo MT, Mussolino C, Goodwin MJ, Cradick TJ, McCaffrey AP, Cathomen T, Scharenberg AM, Joung JK.

Nucleic Acids Res. 2012 Jul;40(12):5560-8. doi: 10.1093/nar/gks179. Epub 2012 Feb 28.

48.

TALE nucleases: tailored genome engineering made easy.

Mussolino C, Cathomen T.

Curr Opin Biotechnol. 2012 Oct;23(5):644-50. doi: 10.1016/j.copbio.2012.01.013. Epub 2012 Feb 17. Review.

PMID:
22342754
49.

DNA-binding activity of adeno-associated virus Rep is required for inverted terminal repeat-dependent complex formation with herpes simplex virus ICP8.

Alex M, Weger S, Mietzsch M, Slanina H, Cathomen T, Heilbronn R.

J Virol. 2012 Mar;86(5):2859-63. doi: 10.1128/JVI.06364-11. Epub 2011 Dec 28.

50.

Selection-independent generation of gene knockout mouse embryonic stem cells using zinc-finger nucleases.

Osiak A, Radecke F, Guhl E, Radecke S, Dannemann N, Lütge F, Glage S, Rudolph C, Cantz T, Schwarz K, Heilbronn R, Cathomen T.

PLoS One. 2011;6(12):e28911. doi: 10.1371/journal.pone.0028911. Epub 2011 Dec 14.

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