Works by Mussolino, Claudio

Results: 29

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

Published in:

Journal of Clinical Medicine, 2019, v. 8, n. 11, p. 1959, doi. 10.3390/jcm8111959

By:

Patsali, Petros;
Mussolino, Claudio;
Ladas, Petros;
Floga, Argyro;
Kolnagou, Annita;
Christou, Soteroula;
Sitarou, Maria;
Antoniou, Michael N.;
Cathomen, Toni;
Lederer, Carsten Werner;
Kleanthous, Marina

Publication type:

Article

Multimodal HLA-I genotype regulation by human cytomegalovirus US10 and resulting surface patterning.

Published in:

eLife, 2024, p. 1, doi. 10.7554/eLife.85560

By:

Gerke, Carolin;
Bauersfeld, Liane;
Schirmeister, Ivo;
Mireisz, Chiara Noemi-Marie;
Oberhardt, Valerie;
Mery, Lea;
Di Wu;
Jürges, Christopher Sebastian;
Spaapen, Robbert M.;
Mussolino, Claudio;
Vu Thuy Khanh Le-Trilling;
Trilling, Mirko;
Dölken, Lars;
Paster, Wolfgang;
Erhard, Florian;
Hofmann, Maike;
Schlosser, Andreas;
Hengel, Hartmut;
Momburg, Frank;
Halenius, Anne

Publication type:

Article

A DNA base-specific sequence interposed between CRX and NRL contributes to RHODOPSIN expression.

Published in:

Scientific Reports, 2024, v. 14, n. 1, p. 1, doi. 10.1038/s41598-024-76664-8

By:

Maritato, Rosa;
Medugno, Alessia;
D'Andretta, Emanuela;
De Riso, Giulia;
Lupo, Mariangela;
Botta, Salvatore;
Marrocco, Elena;
Renda, Mario;
Sofia, Martina;
Mussolino, Claudio;
Bacci, Maria Laura;
Surace, Enrico Maria

Publication type:

Article

DNA Damage: From Threat to Treatment.

Published in:

Cells (2073-4409), 2020, v. 9, n. 7, p. 1665, doi. 10.3390/cells9071665

By:

Carusillo, Antonio;
Mussolino, Claudio

Publication type:

Article

Genome Editing of the SNAI1 Gene in Rhabdomyosarcoma: A Novel Model for Studies of Its Role.

Published in:

Cells (2073-4409), 2020, v. 9, n. 5, p. 1095, doi. 10.3390/cells9051095

By:

Ulman, Aleksandra;
Skrzypek, Klaudia;
Konieczny, Paweł;
Mussolino, Claudio;
Cathomen, Toni;
Majka, Marcin

Publication type:

Article

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

Published in:

Nucleic Acids Research, 2013, v. 41, n. 5, p. e63, doi. 10.1093/nar/gks1446

By:

Holkers, Maarten;
Maggio, Ignazio;
Liu, Jin;
Janssen, Josephine M.;
Miselli, Francesca;
Mussolino, Claudio;
Recchia, Alessandra;
Cathomen, Toni;
Gonçalves, Manuel A. F. V.

Publication type:

Article

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

Published in:

Nucleic Acids Research, 2012, v. 40, n. 13, p. 5560, doi. 10.1093/nar/gks179

By:

Ramirez, Cherie L.;
Certo, Michael T.;
Mussolino, Claudio;
Goodwin, Mathew J.;
Cradick, Thomas J.;
McCaffrey, Anton P.;
Cathomen, Toni;
Scharenberg, Andrew M.;
Joung, J. Keith

Publication type:

Article

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

Published in:

Nucleic Acids Research, 2012, v. 40, n. 12, p. 5560, doi. 10.1093/nar/gks179

By:

Ramirez, Cherie L.;
Certo, Michael T.;
Mussolino, Claudio;
Goodwin, Mathew J.;
Cradick, Thomas J.;
McCaffrey, Anton P.;
Cathomen, Toni;
Scharenberg, Andrew M.;
Joung, J. Keith

Publication type:

Article

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

Published in:

Nucleic Acids Research, 2011, v. 39, n. 21, p. 9283, doi. 10.1093/nar/gkr597

By:

Mussolino, Claudio;
Morbitzer, Robert;
Lütge, Fabienne;
Dannemann, Nadine;
Lahaye, Thomas;
Cathomen, Toni

Publication type:

Article

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

Published in:

PLoS Genetics, 2015, v. 11, n. 5, p. 1, doi. 10.1371/journal.pgen.1005239

By:

Rahman, Shamim H.;
Kuehle, Johannes;
Reimann, Christian;
Mlambo, Tafadzwa;
Alzubi, Jamal;
Maeder, Morgan L.;
Riedel, Heimo;
Fisch, Paul;
Cantz, Tobias;
Rudolph, Cornelia;
Mussolino, Claudio;
Joung, J. Keith;
Schambach, Axel;
Cathomen, Toni

Publication type:

Article

Allele-Specific Disruption of a Common STAT3 Autosomal Dominant Allele Is Not Sufficient to Restore Downstream Signaling in Patient-Derived T Cells.

Published in:

Genes, 2022, v. 13, n. 10, p. N.PAG, doi. 10.3390/genes13101912

By:

König, Saskia;
Fliegauf, Manfred;
Rhiel, Manuel;
Grimbacher, Bodo;
Cornu, Tatjana I.;
Cathomen, Toni;
Mussolino, Claudio

Publication type:

Article

Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets.

Published in:

International Journal of Molecular Sciences, 2024, v. 25, n. 13, p. 7333, doi. 10.3390/ijms25137333

By:

Taghdiri, Maryam;
Mussolino, Claudio

Publication type:

Article

CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells.

Published in:

International Journal of Molecular Sciences, 2024, v. 25, n. 2, p. 985, doi. 10.3390/ijms25020985

By:

Hammad, Ruba;
Alzubi, Jamal;
Rhiel, Manuel;
Chmielewski, Kay O.;
Mosti, Laura;
Rositzka, Julia;
Heugel, Marcel;
Lawrenz, Jan;
Pennucci, Valentina;
Gläser, Birgitta;
Fischer, Judith;
Schambach, Axel;
Moritz, Thomas;
Lachmann, Nico;
Cornu, Tatjana I.;
Mussolino, Claudio;
Schäfer, Richard;
Cathomen, Toni

Publication type:

Article

Chimerization Enables Gene Synthesis and Lentiviral Delivery of Customizable TALE-Based Effectors.

Published in:

International Journal of Molecular Sciences, 2020, v. 21, n. 3, p. 795, doi. 10.3390/ijms21030795

By:

Fang, Yongxing;
Stroukov, Wladislaw;
Cathomen, Toni;
Mussolino, Claudio

Publication type:

Article

From CRISPR screens to circuits: identifying key regulators in T cell activation and state transitions.

Published in:

Signal Transduction & Targeted Therapy, 2025, v. 10, n. 1, p. 1, doi. 10.1038/s41392-025-02200-3

By:

Roman Azcona, Maria Silvia;
Cathomen, Toni;
Mussolino, Claudio

Publication type:

Article

A versatile reporter system for multiplexed screening of effective epigenome editors.

Published in:

Nature Protocols, 2020, v. 15, n. 10, p. 3410, doi. 10.1038/s41596-020-0380-y

By:

Roman Azcona, Maria Silvia;
Fang, Yongxing;
Carusillo, Antonio;
Cathomen, Toni;
Mussolino, Claudio

Publication type:

Article

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

Published in:

2019

By:

Bloom, Kristie;
Kaldine, Haajira;
Cathomen, Toni;
Mussolino, Claudio;
Ely, Abdullah;
Arbuthnot, Patrick

Publication type:

journal article

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

Published in:

EMBO Molecular Medicine, 2011, v. 3, n. 3, p. 118, doi. 10.1002/emmm.201000119

By:

Mussolino, Claudio;
Sanges, Daniela;
Marrocco, Elena;
Bonetti, Ciro;
Di Vicino, Umberto;
Marigo, Valeria;
Auricchio, Alberto;
Meroni, Germana;
Surace, Enrico Maria

Publication type:

Article

Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases.

Published in:

Molecular Therapy, 2013, v. 21, n. 10, p. 1889, doi. 10.1038/mt.2013.170

By:

Bloom, Kristie;
Ely, Abdullah;
Mussolino, Claudio;
Cathomen, Toni;
Arbuthnot, Patrick

Publication type:

Article

Inhibition of Ocular Neovascularization by Hedgehog Blockade.

Published in:

Molecular Therapy, 2006, v. 13, n. 3, p. 573, doi. 10.1016/j.ymthe.2005.10.010

By:

Surace, Enrico M.;
Balaggan, Kamaljit S.;
Tessitore, Alessandra;
Mussolino, Claudio;
Cotugno, Gabriella;
Bonetti, Ciro;
Vitale, Aniello;
Ali, Robin R.;
Auricchio, Alberto

Publication type:

Article

Preclinical Evaluation of a Novel TALEN Targeting CCR5 Confirms Efficacy and Safety in Conferring Resistance to HIV‐1 Infection.

Published in:

Biotechnology Journal, 2021, v. 16, n. 1, p. 1, doi. 10.1002/biot.202000023

By:

Romito, Marianna;
Juillerat, Alexandre;
Kok, Yik Lim;
Hildenbeutel, Markus;
Rhiel, Manuel;
Andrieux, Geoffroy;
Geiger, Johannes;
Rudolph, Carsten;
Mussolino, Claudio;
Duclert, Aymeric;
Metzner, Karin J.;
Duchateau, Philippe;
Cathomen, Toni;
Cornu, Tatjana I.

Publication type:

Article

Gene Therapy Getting Personal: Mutation-Specific Editing and Gene Addition Strategies for β-Thalassaemia.

Published in:

2019

By:

Lederer, Carsten W.;
Patsali, Petros;
Mussolino, Claudio;
Turchiano, Giandomenico;
Papasavva, Panayiota;
Romito, Marianna;
Stephanou, Coralea;
Cornu, Tatjana I.;
Kolnagou, Annita;
Christou, Soteroula;
Sitarou, Maria;
Antoniou, Mike N.;
Cathomen, Toni;
Kleanthous, Marina

Publication type:

Abstract

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

Published in:

Nucleic Acids Research, 2018, v. 46, n. 9, p. 4456, doi. 10.1093/nar/gky171

By:

Mlambo, Tafadzwa;
Nitsch, Sandra;
Hildenbeutel, Markus;
Romito, Marianna;
Müller, Maximilian;
Bossen, Claudia;
Diederichs, Sven;
Cornu, Tatjana I;
Cathomen, Toni;
Mussolino, Claudio

Publication type:

Article

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

Published in:

Nucleic Acids Research, 2014, v. 42, n. 10, p. 6762, doi. 10.1093/nar/gku305

By:

Mussolino, Claudio;
Alzubi, Jamal;
Fine, Eli J.;
Morbitzer, Robert;
Cradick, Thomas J.;
Lahaye, Thomas;
Bao, Gang;
Cathomen, Toni

Publication type:

Article

On target? Tracing zinc-finger-nuclease specificity.

Published in:

2011

By:

Mussolino, Claudio;
Cathomen, Toni

Publication type:

Opinion

TALEN mediated gene editing in a mouse model of Fanconi anemia.

Published in:

Scientific Reports, 2020, v. 10, n. 1, p. 1, doi. 10.1038/s41598-020-63971-z

By:

Pino-Barrio, Maria José;
Giménez, Yari;
Villanueva, Mariela;
Hildenbeutel, Marcus;
Sánchez-Dominguez, Rebeca;
Rodríguez-Perales, Sandra;
Pujol, Roser;
Surrallés, Jordi;
Río, Paula;
Cathomen, Toni;
Mussolino, Claudio;
Bueren, Juan Antonio;
Navarro, Susana

Publication type:

Article

Fine-Tuning Homology-Directed Repair (HDR) for Precision Genome Editing: Current Strategies and Future Directions.

Published in:

International Journal of Molecular Sciences, 2025, v. 26, n. 9, p. 4067, doi. 10.3390/ijms26094067

By:

Haider, Sibtain;
Mussolino, Claudio

Publication type:

Article

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

Published in:

Scientific Reports, 2016, p. 38198, doi. 10.1038/srep38198

By:

Eggenschwiler, Reto;
Moslem, Mohsen;
Fráguas, Mariane Serra;
Galla, Melanie;
Papp, Oliver;
Naujock, Maximilian;
Fonfara, Ines;
Gensch, Ingrid;
Wähner, Annabell;
Beh-Pajooh, Abbas;
Mussolino, Claudio;
Tauscher, Marcel;
Steinemann, Doris;
Wegner, Florian;
Petri, Susanne;
Schambach, Axel;
Charpentier, Emmanuelle;
Cathomen, Toni;
Cantz, Tobias

Publication type:

Article

RNA guides genome engineering.

Published in:

Nature Biotechnology, 2013, v. 31, n. 3, p. 208, doi. 10.1038/nbt.2527

By:

Mussolino, Claudio;
Cathomen, Toni

Publication type:

Article