Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates von Chuah, Marinee K L, Ma, Ling, VandenDriessche, Thierry, Manoj, Namitha, Samara-Kuko, Ermira, Grzela, Dawid P, Schmitt, Andrea, Pryputniewicz, Diana, Fletcher, Bradley, Jerchow, Boris, Mátés, Lajos, Matrai, Janka, Gysemans, Conny, Becker, Katja, Miskey, Csaba, Belay, Eyayu, Acosta-Sanchez, Abel, Ivics, Zoltán, Izsvák, Zsuzsanna

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Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants von Weinmann, Jonas, Weis, Sabrina, Sippel, Josefine, Tulalamba, Warut, Remes, Anca, El Andari, Jihad, Herrmann, Anne-Kathrin, Pham, Quang H., Borowski, Christopher, Hille, Susanne, Schönberger, Tanja, Frey, Norbert, Lenter, Martin, VandenDriessche, Thierry, Müller, Oliver J., Chuah, Marinee K., Lamla, Thorsten, Grimm, Dirk

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Hepatocyte‐targeted expression by integrase‐defective lentiviral vectors induces antigen‐specific tolerance in mice with low genotoxic risk von Mátrai, Janka, Cantore, Alessio, Bartholomae, Cynthia C., Annoni, Andrea, Wang, Wei, Acosta‐Sanchez, Abel, Samara‐Kuko, Ermira, De Waele, Liesbeth, Ma, Ling, Genovese, Pietro, Damo, Martina, Arens, Anne, Goudy, Kevin, Nichols, Timothy C., von Kalle, Christof, L. Chuah, Marinee K., Roncarolo, Maria Grazia, Schmidt, Manfred, VandenDriessche, Thierry, Naldini, Luigi

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Recent Advances in Lentiviral Vector Development and Applications von Mátrai, Janka, Chuah, Marinee KL, VandenDriessche, Thierry

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Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation von Rincon, Melvin Y, VandenDriessche, Thierry, Chuah, Marinee K

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UNIVERSAL ALLOGENEIC CAR T CELLS ENGINEERED WITH SLEEPING BEAUTY TRANSPOSONS AND CRISPR/CAS9 FOR CANCER IMMUNOTHERAPY von Tipanee, Jaitip, Samara-Kuko, Ermira, Gevaert, Thierry, Chuah, Marinee K., VandenDriessche, Thierry

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Codon optimization of human factor VIII cDNAs leads to high-level expression von Ward, Natalie J., Buckley, Suzanne M.K., Waddington, Simon N., VandenDriessche, Thierry, Chuah, Marinee K.L., Nathwani, Amit C., McIntosh, Jenny, Tuddenham, Edward G.D., Kinnon, Christine, Thrasher, Adrian J., McVey, John H.

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Transposons: Moving Forward from Preclinical Studies to Clinical Trials von Tipanee, Jaitip, VandenDriessche, Thierry, Chuah, Marinee K

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Hemophilia Gene Therapy: The End of the Beginning? von De Wolf, Dries, Singh, Kshitiz, Chuah, Marinee K., VandenDriessche, Thierry

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Preclinical and clinical advances in transposon-based gene therapy von Tipanee, Jaitip, Chai, Yoke Chin, VandenDriessche, Thierry, Chuah, Marinee K

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Hemophilia “A” gene therapy: Lost in translation von VandenDriessche, Thierry, Chuah, Marinee K.

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Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells von VandenDriessche, Thierry, Ivics, Zoltán, Izsvák, Zsuzsanna, Chuah, Marinee K.L.

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Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes von Dastidar, Sumitava, Majumdar, Debanjana, Tipanee, Jaitip, Singh, Kshitiz, Klein, Arnaud F., Furling, Denis, Chuah, Marinee K., VandenDriessche, Thierry

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Hemophilia Gene Therapy: Ready for Prime Time? von VandenDriessche, Thierry, Chuah, Marinee K

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Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy von Di Matteo, Mario, Samara-Kuko, Emira, Ward, Natalie J, Waddingon, Simon N, McVey, John H, Chuah, Marinee KL, VandenDriessche, Thierry

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Hyperactive Factor IX Padua: A Game-Changer for Hemophilia Gene Therapy von VandenDriessche, Thierry, Chuah, Marinee K.

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Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9 von Singh, Kshitiz, Evens, Hanneke, Nair, Nisha, Rincón, Melvin Y., Sarcar, Shilpita, Samara-Kuko, Ermira, Chuah, Marinee K., VandenDriessche, Thierry

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Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs von Quattrocelli, Mattia, Palazzolo, Giacomo, Floris, Giuseppe, Schöffski, Patrick, Anastasia, Luigi, Orlacchio, Aldo, Vandendriessche, Thierry, Chuah, Marinee KL, Cossu, Giulio, Verfaillie, Catherine, Sampaolesi, Maurilio

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Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells von Dastidar, Sumitava, Ardui, Simon, Singh, Kshitiz, Majumdar, Debanjana, Nair, Nisha, Fu, Yanfang, Reyon, Deepak, Samara, Ermira, Gerli, Mattia F M, Klein, Arnaud F, De Schrijver, Wito, Tipanee, Jaitip, Seneca, Sara, Tulalamba, Warut, Wang, Hui, Chai, Yoke Chin, In't Veld, Peter, Furling, Denis, Tedesco, Francesco Saverio, Vermeesch, Joris R, Joung, J Keith, Chuah, Marinee K, VandenDriessche, Thierry

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Comparative Analysis of Transposable Element Vector Systems in Human Cells von Grabundzija, Ivana, Irgang, Markus, Mátés, Lajos, Belay, Eyayu, Matrai, Janka, Gogol-Döring, Andreas, Kawakami, Koichi, Chen, Wei, Ruiz, Patricia, Chuah, Marinee K.L., VandenDriessche, Thierry, Izsvák, Zsuzsanna, Ivics, Zoltán

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