Search Results - "Chuah, Marinee K. L."

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates by 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

“…Zsuzsanna Izsvák and colleagues report the generation of a hyperactive version of the Sleeping Beauty transposase that supports efficient and stable gene…”
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Hepatocyte‐targeted expression by integrase‐defective lentiviral vectors induces antigen‐specific tolerance in mice with low genotoxic risk by 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

“…Lentiviral vectors are attractive tools for liver‐directed gene therapy because of their capacity for stable gene expression and the lack of preexisting…”
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Codon optimization of human factor VIII cDNAs leads to high-level expression by 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.

“…Gene therapy for hemophilia A would be facilitated by development of smaller expression cassettes encoding factor VIII (FVIII), which demonstrate improved…”
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Emerging potential of transposons for gene therapy and generation of induced pluripotent stem cells by VandenDriessche, Thierry, Ivics, Zoltán, Izsvák, Zsuzsanna, Chuah, Marinee K.L.

“…Effective gene therapy requires robust delivery of the desired genes into the relevant target cells, long-term gene expression, and minimal risks of secondary…”
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Hyperactive PiggyBac Transposons for Sustained and Robust Liver-targeted Gene Therapy by Di Matteo, Mario, Samara-Kuko, Emira, Ward, Natalie J, Waddingon, Simon N, McVey, John H, Chuah, Marinee KL, VandenDriessche, Thierry

“…The development of robust nonviral vectors could facilitate clinical gene therapy applications and may overcome some of the immune complications of viral…”
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Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs by Quattrocelli, Mattia, Palazzolo, Giacomo, Floris, Giuseppe, Schöffski, Patrick, Anastasia, Luigi, Orlacchio, Aldo, Vandendriessche, Thierry, Chuah, Marinee KL, Cossu, Giulio, Verfaillie, Catherine, Sampaolesi, Maurilio

“…Mesoangioblasts (MABs) are a subset of muscle‐derived pericytes able to restore dystrophic phenotype in mice and dogs. However, their lifespan is limited and…”
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Recent Advances in Lentiviral Vector Development and Applications by Mátrai, Janka, Chuah, Marinee KL, VandenDriessche, Thierry

“…Lentiviral vectors (LVs) have emerged as potent and versatile vectors for ex vivo or in vivo gene transfer into dividing and nondividing cells. Robust…”
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Preclinical and clinical progress in hemophilia gene therapy by Mátrai, Janka, Chuah, Marinee KL, VandenDriessche, Thierry

“…PURPOSE OF REVIEWHemophilia A and B are attractive target diseases for gene therapy, as stable expression of coagulation factor VIII and IX may correct the…”
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Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors by Izsvák, Zsuzsanna, Chuah, Marinee K.L., VandenDriessche, Thierry, Ivics, Zoltán

“…Transposable elements can be considered as natural, non-viral gene delivery vehicles capable of efficient genomic insertion. The plasmid-based transposon…”
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Restoration of Plasma von Willebrand Factor Deficiency Is Sufficient to Correct Thrombus Formation After Gene Therapy for Severe von Willebrand Disease by De Meyer, Simon F, Vandeputte, Nele, Pareyn, Inge, Petrus, Inge, Lenting, Peter J, Chuah, Marinee K.L, VandenDriessche, Thierry, Deckmyn, Hans, Vanhoorelbeke, Karen

“…OBJECTIVE—Gene therapy for severe von Willebrand disease (vWD) seems an interesting treatment alternative with long-term therapeutic potential. We investigated…”
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Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo by VandenDriessche, Thierry, Thorrez, Lieven, Naldini, Luigi, Follenzi, Antonia, Moons, Lieve, Berneman, Zwi, Collen, Desire, Chuah, Marinee K.L.

“…High-titer self-inactivating human immunodeficiency virus type-1 (HIV-1)–based vectors expressing the green fluorescent protein reporter gene that contained…”
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Nanoparticles for the delivery of genes and drugs to human hepatocytes by Kuroda, Shun'ichi, Yamada, Tadanori, Iwasaki, Yasushi, Tada, Hiroko, Iwabuki, Hidehiko, Chuah, Marinee KL, VandenDriessche, Thierry, Fukuda, Hideki, Kondo, Akihiko, Ueda, Masakazu, Seno, Masaharu, Tanizawa, Katsuyuki

“…Hepatitis B virus envelope L particles form hollow nanoparticles displaying a peptide that is indispensable for liver-specific infection by hepatitis B virus…”
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Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors by Chuah, Marinee K.L., Schiedner, Gudrun, Thorrez, Lieven, Brown, Brian, Johnston, Marion, Gillijns, Veerle, Hertel, Sabine, Van Rooijen, Nico, Lillicrap, David, Collen, Désiré, VandenDriessche, Thierry, Kochanek, Stefan

“…High-capacity adenoviral (HC-Ad) vectors expressing B-domain–deleted human or canine factor VIII from different liver-specific promoters were evaluated for…”
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Efficient Lentiviral Transduction and Improved Engraftment of Human Bone Marrow Mesenchymal Cells by Van Damme, An, Thorrez, Lieven, Ma, Ling, Vandenburgh, Herman, Eyckmans, Jeroen, Dell'Accio, Francesco, De Bari, Cosimo, Luyten, Frank, Lillicrap, David, Collen, Désiré, VandenDriessche, Thierry, Chuah, Marinee K.L.

“…Human bone marrow (BM) mesenchymal stem/progenitor cells are potentially attractive targets for ex vivo gene therapy. The potential of lentiviral vectors for…”
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Comparative Analysis of Transposable Element Vector Systems in Human Cells by 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

“…Transposon-based gene vectors have become indispensable tools in vertebrate genetics for applications ranging from insertional mutagenesis and transgenesis in…”
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Gene therapy for hemophilia by Chuah, Marinee K. L., Collen, Desire, VandenDriessche, Thierry

“…Hemophilia A and B are X‐chromosome linked recessive bleeding disorders that result from a deficiency in factor VIII (FVIII) and factor IX (FIX) respectively…”
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Novel Hyperactive Transposons for Genetic Modification of Induced Pluripotent and Adult Stem Cells: A Nonviral Paradigm for Coaxed Differentiation by Belay, Eyayu, Mátrai, Janka, Acosta‐Sanchez, Abel, Ma, Ling, Quattrocelli, Mattia, Mátés, Lajos, Sancho‐Bru, Pau, Geraerts, Martine, Yan, Bing, Vermeesch, Joris, Rincón, Melvin Yesid, Samara‐Kuko, Ermira, Ivics, Zoltán, Verfaillie, Catherine, Sampaolesi, Maurilio, Izsvák, Zsuzsanna, VandenDriessche, Thierry, Chuah, Marinee K. L.

“…Adult stem cells and induced pluripotent stem cells (iPS) hold great promise for regenerative medicine. The development of robust nonviral approaches for stem…”
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Transposon-mediated gene transfer into adult and induced pluripotent stem cells by Belay, Eyayu, Dastidar, Sumitava, VandenDriessche, Thierry, Chuah, Marinee K L

“…Transposon technology is a particularly attractive non-viral gene delivery paradigm that allows for efficient genomic integration into a variety of different…”
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Moving gene therapy forward with mobile DNA by VandenDriessche, Thierry, Chuah, Marinee K L

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PiggyBac toolbox by Di Matteo, Mario, Mátrai, Janka, Belay, Eyayu, Firdissa, Tewodros, Vandendriessche, Thierry, Chuah, Marinee K L

“…The PiggyBac (PB) transposon system was originally derived from the cabbage looper moth Trichoplusia ni and represents one of the most promising transposon…”
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