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Items: 32


The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements.

Patsali P, Mussolino C, Ladas P, Floga A, Kolnagou A, Christou S, Sitarou M, Antoniou MN, Cathomen T, Lederer CW, Kleanthous M.

J Clin Med. 2019 Nov 13;8(11). pii: E1959. doi: 10.3390/jcm8111959.


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.



Mussolino C.

Stem Cells Dev. 2019 Jun 1;28(11):714. doi: 10.1089/scd.2019.0078. Epub 2019 May 13. No abstract available.


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.


Fast and Quantitative Identification of Ex Vivo Precise Genome Targeting-Induced Indel Events by IDAA.

König S, Yang Z, Wandall HH, Mussolino C, Bennett EP.

Methods Mol Biol. 2019;1961:45-66. doi: 10.1007/978-1-4939-9170-9_4.


Precise Epigenome Editing on the Stage: A Novel Approach to Modulate Gene Expression.

Mussolino C.

Epigenet Insights. 2018 Dec 13;11:2516865718818838. doi: 10.1177/2516865718818838. eCollection 2018.


Erratum to: Delivery of Designer Epigenome Modifiers into Primary Human T Cells.

Mlambo T, Romito M, Cornu TI, Mussolino C.

Methods Mol Biol. 2018;1767:E1. doi: 10.1007/978-1-4939-7774-1_26. No abstract available.


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.


Delivery of Designer Epigenome Modifiers into Primary Human T Cells.

Mlambo T, Romito M, Cornu TI, Mussolino C.

Methods Mol Biol. 2018;1767:189-203. doi: 10.1007/978-1-4939-7774-1_9. Erratum in: Methods Mol Biol. 2018;1767:E1.


Generation of TALE-Based Designer Epigenome Modifiers.

Nitsch S, Mussolino C.

Methods Mol Biol. 2018;1767:89-109. doi: 10.1007/978-1-4939-7774-1_4.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


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.


RNA guides genome engineering.

Mussolino C, Cathomen T.

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


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.


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.


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.


On target? Tracing zinc-finger-nuclease specificity.

Mussolino C, Cathomen T.

Nat Methods. 2011 Aug 30;8(9):725-6. doi: 10.1038/nmeth.1680.


A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity.

Mussolino C, Morbitzer R, Lütge F, Dannemann N, Lahaye T, Cathomen T.

Nucleic Acids Res. 2011 Nov;39(21):9283-93. doi: 10.1093/nar/gkr597. Epub 2011 Aug 3.


AAV-mediated photoreceptor transduction of the pig cone-enriched retina.

Mussolino C, della Corte M, Rossi S, Viola F, Di Vicino U, Marrocco E, Neglia S, Doria M, Testa F, Giovannoni R, Crasta M, Giunti M, Villani E, Lavitrano M, Bacci ML, Ratiglia R, Simonelli F, Auricchio A, Surace EM.

Gene Ther. 2011 Jul;18(7):637-45. doi: 10.1038/gt.2011.3. Epub 2011 Mar 17.


Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa.

Mussolino C, Sanges D, Marrocco E, Bonetti C, Di Vicino U, Marigo V, Auricchio A, Meroni G, Surace EM.

EMBO Mol Med. 2011 Mar;3(3):118-28. doi: 10.1002/emmm.201000119. Epub 2011 Jan 26.


Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors.

Allocca M, Mussolino C, Garcia-Hoyos M, Sanges D, Iodice C, Petrillo M, Vandenberghe LH, Wilson JM, Marigo V, Surace EM, Auricchio A.

J Virol. 2007 Oct;81(20):11372-80. Epub 2007 Aug 15.


Inhibition of ocular neovascularization by hedgehog blockade.

Surace EM, Balaggan KS, Tessitore A, Mussolino C, Cotugno G, Bonetti C, Vitale A, Ali RR, Auricchio A.

Mol Ther. 2006 Mar;13(3):573-9. Epub 2005 Dec 15.

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