Clinical genome editing to treat sickle cell disease—A brief update

por Zarghamian, Parinaz, Klermund, Julia, Cathomen, Toni
Publicado 2023

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

por Fang, Yongxing, Stroukov, Wladislaw, Cathomen, Toni, Mussolino, Claudio
Publicado 2020

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

por Bloom, Kristie, Ely, Abdullah, Mussolino, Claudio, Cathomen, Toni, Arbuthnot, Patrick
Publicado 2013

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

por Aushev, Magomet, Koller, Ulrich, Mussolino, Claudio, Cathomen, Toni, Reichelt, Julia
Publicado 2017

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

por Mussolino, Claudio, Morbitzer, Robert, Lütge, Fabienne, Dannemann, Nadine, Lahaye, Thomas, Cathomen, Toni
Publicado 2011

A novel zinc-finger nuclease platform with a sequence-specific cleavage module

por Schierling, Benno, Dannemann, Nadine, Gabsalilow, Lilia, Wende, Wolfgang, Cathomen, Toni, Pingoud, Alfred
Publicado 2012

TALE-PvuII Fusion Proteins – Novel Tools for Gene Targeting

por Yanik, Mert, Alzubi, Jamal, Lahaye, Thomas, Cathomen, Toni, Pingoud, Alfred, Wende, Wolfgang
Publicado 2013

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

por Bednarski, Christien, Tomczak, Katja, vom Hövel, Beate, Weber, Wolf-Michael, Cathomen, Toni
Publicado 2016

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

por Kuhn, Alexandra, Ackermann, Mania, Mussolino, Claudio, Cathomen, Toni, Lachmann, Nico, Moritz, Thomas
Publicado 2017

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

por Bloom, Kristie, Kaldine, Haajira, Cathomen, Toni, Mussolino, Claudio, Ely, Abdullah, Arbuthnot, Patrick
Publicado 2019

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

por Ulman, Aleksandra, Skrzypek, Klaudia, Konieczny, Paweł, Mussolino, Claudio, Cathomen, Toni, Majka, Marcin
Publicado 2020

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

por Mock, Ulrike, Riecken, Kristoffer, Berdien, Belinda, Qasim, Waseem, Chan, Emma, Cathomen, Toni, Fehse, Boris
Publicado 2014

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

por Alzubi, Jamal, Pallant, Celeste, Mussolino, Claudio, Howe, Steven J., Thrasher, Adrian J., Cathomen, Toni
Publicado 2017

Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells

por Mosti, Laura, Langner, Lukas M., Chmielewski, Kay O., Arbuthnot, Patrick, Alzubi, Jamal, Cathomen, Toni
Publicado 2021

Retargeting transposon insertions by the adeno-associated virus Rep protein

por Ammar, Ismahen, Gogol-Döring, Andreas, Miskey, Csaba, Chen, Wei, Cathomen, Toni, Izsvák, Zsuzsanna, Ivics, Zoltán
Publicado 2012

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

por Bobis-Wozowicz, Sylwia, Galla, Melanie, Alzubi, Jamal, Kuehle, Johannes, Baum, Christopher, Schambach, Axel, Cathomen, Toni
Publicado 2014

Assessing the differential impact of chronic CMV and treated HIV infection on CD8+ T-cell differentiation in a matched cohort study: is CMV the key?

por Mueller, Matthias C., Kern, Winfried V., Usadel, Susanne, Pauly, Marie-Christin, Cathomen, Toni, Salzer, Ulrich
Publicado 2021

Improved Specificity and Safety of Anti-Hepatitis B Virus TALENs Using Obligate Heterodimeric FokI Nuclease Domains

por Smith, Tiffany, Singh, Prashika, Chmielewski, Kay Ole, Bloom, Kristie, Cathomen, Toni, Arbuthnot, Patrick, Ely, Abdullah
Publicado 2021

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

por König, Saskia, Fliegauf, Manfred, Rhiel, Manuel, Grimbacher, Bodo, Cornu, Tatjana I., Cathomen, Toni, Mussolino, Claudio
Publicado 2022

T-CAST: An optimized CAST-Seq pipeline for TALEN confirms superior safety and efficacy of obligate-heterodimeric scaffolds

por Rhiel, Manuel, Geiger, Kerstin, Andrieux, Geoffroy, Rositzka, Julia, Boerries, Melanie, Cathomen, Toni, Cornu, Tatjana I.
Publicado 2023