Search Results - "Samara‐Kuko, Ermira"

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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Validation of miR-20a as a Tumor Suppressor Gene in Liver Carcinoma Using Hepatocyte-Specific Hyperactive piggyBac Transposons by Tipanee, Jaitip, Di Matteo, Mario, Tulalamba, Warut, Samara-Kuko, Ermira, Keirsse, Jiri, Van Ginderachter, Jo A., Chuah, Marinee Khim, VandenDriessche, Thierry

“…We established a semi-high-throughput in vivo screening platform using hyperactive piggyBac (hyPB) transposons (designated as PB-miR) to identify microRNAs…”
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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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UNIVERSAL ALLOGENEIC CAR T CELLS ENGINEERED WITH SLEEPING BEAUTY TRANSPOSONS AND CRISPR/CAS9 FOR CANCER IMMUNOTHERAPY by Tipanee, Jaitip, Samara-Kuko, Ermira, Gevaert, Thierry, Chuah, Marinee K., VandenDriessche, Thierry

“…Allogeneic CD19-specific chimeric antigen receptor (CAR) T cells with inactivated donor T cell receptor (TCR) expression can be used as an ‘off-the-shelf’…”
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Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9 by Singh, Kshitiz, Evens, Hanneke, Nair, Nisha, Rincón, Melvin Y., Sarcar, Shilpita, Samara-Kuko, Ermira, Chuah, Marinee K., VandenDriessche, Thierry

“…In vivo tissue-specific genome editing at the desired loci is still a challenge. Here, we report that AAV9-delivery of truncated guide RNAs (gRNAs) and Cas9…”
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Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua by Nair, Nisha, De Wolf, Dries, Nguyen, Phuong Anh, Pham, Quang Hong, Samara-Kuko, Ermira, Landau, Jeff, Blouse, Grant E., Chuah, Marinee K., Vanden Driessche, Thierry

“…Sustained expression of therapeutic factor IX (FIX) levels has been achieved after adeno-associated viral (AAV) vector-based gene therapy in patients with…”
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piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts by Loperfido, Mariana, Jarmin, Susan, Dastidar, Sumitava, Di Matteo, Mario, Perini, Ilaria, Moore, Marc, Nair, Nisha, Samara-Kuko, Ermira, Athanasopoulos, Takis, Tedesco, Francesco Saverio, Dickson, George, Sampaolesi, Maurilio, VandenDriessche, Thierry, Chuah, Marinee K

“…Duchenne muscular dystrophy (DMD) is a genetic neuromuscular disorder caused by the absence of dystrophin. We developed a novel gene therapy approach based on…”
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Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy by Rincon, Melvin Y, Sarcar, Shilpita, Danso-Abeam, Dina, Keyaerts, Marleen, Matrai, Janka, Samara-Kuko, Ermira, Acosta-Sanchez, Abel, Athanasopoulos, Takis, Dickson, George, Lahoutte, Tony, De Bleser, Pieter, VandenDriessche, Thierry, Chuah, Marinee K

“…Gene therapy is a promising emerging therapeutic modality for the treatment of cardiovascular diseases and hereditary diseases that afflict the heart. Hence,…”
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Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates by Chuah, Marinee K, Petrus, Inge, De Bleser, Pieter, Le Guiner, Caroline, Gernoux, Gwladys, Adjali, Oumeya, Nair, Nisha, Willems, Jessica, Evens, Hanneke, Rincon, Melvin Y, Matrai, Janka, Di Matteo, Mario, Samara-Kuko, Ermira, Yan, Bing, Acosta-Sanchez, Abel, Meliani, Amine, Cherel, Ghislaine, Blouin, Véronique, Christophe, Olivier, Moullier, Philippe, Mingozzi, Federico, VandenDriessche, Thierry

“…The robustness and safety of liver-directed gene therapy can be substantially improved by enhancing expression of the therapeutic transgene in the liver. To…”
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120. Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9 by Singh, Kshitiz, Evens, Hanneke, Rincón, Melvin, Nair, Nisha, Sarcar, Shilpita, Samara-Kuko, Ermira, Chuah, Marinee K., VandenDriessche, Thierry

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628. Transposons Expressing Full-Length Human Dystrophin Enable Genetic Correction of Dystrophic Mesoangioblasts and iPS-Derived Mesoangioblast-Like Cells by Loperfido, Mariana, Jarmin, Susan, Dastidar, Sumitava, Di Matteo, Mario, Perini, Ilaria, Moore, Marc, Nair, Nisha, Samara-Kuko, Ermira, Athanasopoulos, Takis, Tedesco, Francesco Saverio, Dickson, George, Sampaolesi, Maurilio, VandenDriessche, Thierry, Chuah, Marinee K.

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