Survival estimates of childhood malignancies treated at the Mexican telethon pediatric oncology hospital by Joel Monárrez‐Espino, Lourdes Romero‐Rodriguez, Gabriela Escamilla‐Asiain, Andrea Ellis‐Irigoyen, María del Pilar Cubría‐Juárez, Douglas Sematimba, Carlos Rodríguez‐Galindo, Lourdes Vega‐Vega

“…While survival rates have been reported in various Mexican studies, no data is available from the Telethon Pediatric Oncology Hospital‐HITO, a nonprofit private institution specialized exclusively in comprehensive pediatric oncology care in the country that closely follows high‐income countries' advanced standards of cancer care. …”
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The alliance between genetic biobanks and patient organisations: the experience of the telethon network of genetic biobanks by Chiara Baldo, Lorena Casareto, Alessandra Renieri, Giuseppe Merla, Barbara Garavaglia, Stefano Goldwurm, Elena Pegoraro, Maurizio Moggio, Marina Mora, Luisa Politano, Luca Sangiorgi, Raffaella Mazzotti, Valeria Viotti, Ilaria Meloni, Maria Teresa Pellico, Chiara Barzaghi, Chiuhui Mary Wang, Lucia Monaco, Mirella Filocamo

“…In this context, Genetic Biobanks are critical to developing basic, translational and clinical research on RDs. The Telethon Network of Genetic Biobanks (TNGB) is aware of the importance of biobanking as a service for patients and has started a dialogue with RD-Patient Organisations via promotion of dedicated meetings and round-tables, as well as by including their representatives on the TNGB Advisory Board. …”
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Disaster Handling through Volunteering and Charitable Donation: The Roles of South Korean Telethon, Involvement, and Collective-Efficacy by Bumsub Jin

Subjects: “…volunteering; charitable donation; telethon; involvement; collective-efficacy…”
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Patient‐driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis by Lucia Monaco, Lucia Faccio

“…The recent approval of Strimvelis, the first ex vivo gene therapy to gain marketing authorization (Schimmer & Breazzano, ), has drawn attention to Fondazione Telethon, the Italian charity that played a pivotal role in this effort. …”
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“Gane usted y ayude a la Teletón”: mecanismos neoliberales en la gestión del bienestar by Mónica Humeres

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P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up* by Francesca Fumagalli, Valeria Calbi, Fabiola De Mattia, Alberto Zambon, Vera Gallo, Salvatore Recupero, Elena Sophia Fratini, Alessia Ippolito, Francesca Ciotti, Maddalena Fraschini, Marina Sarzana, Stefano Scarparo, Eugenio Montini, Sara Locatelli, Marcella Facchini, Alessandra Clerici, Francesco Morena, Sabata Martino, Muska Yarzi, Sean Moro, Andrew Shenker, Jean Brooks, Alan Richardson, Leslie Meltzer, Laura Campbell, Alessandro Aiuti

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S250: UNVEILING THE BIOLOGICAL ROLE OF PERIPHERAL BLOOD HUMAN CIRCULATING HEMATOPOIETIC STEM AND PROGENITOR CELLS by Pamela Quaranta, Luca Basso-Ricci, Raisa Jofra Hernandez, Matteo Maria Naldini, Matteo Barcella, Guido Pacini, Carlo Pietrasanta, Lorenza Pugni, Giulia Pais, Fabrizio Benedicenti, Stefania Giannelli, Ilaria Monti, Silvia Darin, Graziano Barera, Francesca Tucci, Francesca Ferrua, Valeria Calbi, Bernhard Gentner, Raffaella DI Micco, Eugenio Montini, Andrea Calabria, Ivan Merelli, Fabio Mosca, Maria Ester Bernardo, Maria Pia Cicalese, Alessandro Aiuti, Scala Serena

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The VersaLive platform enables microfluidic mammalian cell culture for versatile applications by Giovanni Marco Nocera, Gaetano Viscido, Stefania Criscuolo, Simona Brillante, Fabrizia Carbone, Leopoldo Staiano, Sabrina Carrella, Diego di Bernardo

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miR‐181a/b downregulation: a mutation‐independent therapeutic approach for inherited retinal diseases by Sabrina Carrella, Martina Di Guida, Simona Brillante, Davide Piccolo, Ludovica Ciampi, Irene Guadagnino, Jorge Garcia Piqueras, Mariateresa Pizzo, Elena Marrocco, Marta Molinari, Georgios Petrogiannakis, Sara Barbato, Yulia Ezhova, Alberto Auricchio, Brunella Franco, Elvira De Leonibus, Enrico Maria Surace, Alessia Indrieri, Sandro Banfi

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P1373: UNCOVERING UPSIDES AND PITFALLS OF BASE AND PRIME EDITING IN HEMATOPOIETIC STEM CELLS by Martina Fiumara, Samuele Ferrari, Attya Omer-Javed, Stefano Beretta, Luisa Albano, Daniele Canarutto, Angelica Varesi, Chiara Gaddoni, Chiara Brombin, Federica Cugnata, Erika Zonari, Matteo Maria Naldini, Matteo Barcella, Bernhard Gentner, Ivan Merelli, Luigi Naldini

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The Investigation of Health-Related Topics on TikTok: A Descriptive Study Protocol by Joelie Mandzufas, Jeremiah Ayalde, Daniel Ta, Emily Munro, Rigel Paciente, Emmanuel Philip Pranoto, Kaelyn King, Kelly How, Alanna Sincovich, Mary Brushe, Nicole Wickens, Gabriella Wells, Alix Woolard, Melinda Edmunds, Hannah Thomas, Gina S. A. Trapp, Karen Lombardi

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Western Australian adolescent emotional wellbeing during the COVID-19 pandemic in 2020 by H. M. Thomas, K. C. Runions, L. Lester, K. Lombardi, M. Epstein, J. Mandzufas, T. Barrow, S. Ang, A. Leahy, M. Mullane, A. Whelan, J. Coffin, F. Mitrou, S. R. Zubrick, A. C. Bowen, P. W. Gething, D. Cross

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Characterization of mesenchymal stem cells in pre-B acute lymphoblastic leukemia by Anastasia M. Hughes, Anastasia M. Hughes, Vincent Kuek, Vincent Kuek, Vincent Kuek, Joyce Oommen, Grace-Alyssa Chua, Maria van Loenhout, Sebastien Malinge, Sebastien Malinge, Rishi S. Kotecha, Rishi S. Kotecha, Rishi S. Kotecha, Rishi S. Kotecha, Laurence C. Cheung, Laurence C. Cheung, Laurence C. Cheung

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S255: CELLULAR SENESCENCE AND INFLAMMATORY PROGRAMS ARE UNINTENDED CONSEQUENCES OF CRISPR-CAS9 GENE EDITING IN HEMATOPOIETIC STEM AND PROGENITOR CELLS by Anastasia Conti, Teresa Tavella, Rosaria De Marco, Federico Midena, Samuele Ferrari, Stefano Beretta, Chiara Brombin, Ivan Merelli, Luigi Naldini, Raffaella DI Micco

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Loss of the batten disease protein CLN3 leads to mis-trafficking of M6PR and defective autophagic-lysosomal reformation by Alessia Calcagni’, Leopoldo Staiano, Nicolina Zampelli, Nadia Minopoli, Niculin J. Herz, Giuseppe Di Tullio, Tuong Huynh, Jlenia Monfregola, Alessandra Esposito, Carmine Cirillo, Aleksandar Bajic, Mahla Zahabiyon, Rachel Curnock, Elena Polishchuk, Luke Parkitny, Diego Luis Medina, Nunzia Pastore, Peter J. Cullen, Giancarlo Parenti, Maria Antonietta De Matteis, Paolo Grumati, Andrea Ballabio

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Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo by Claudia Asperti, Daniele Canarutto, Simona Porcellini, Francesca Sanvito, Francesca Cecere, Valentina Vavassori, Samuele Ferrari, Elisabetta Rovelli, Luisa Albano, Aurelien Jacob, Lucia Sergi Sergi, Elisa Montaldo, Francesca Ferrua, Luis Ignacio González-Granado, Vassilios Lougaris, Raffaele Badolato, Andrea Finocchi, Anna Villa, Marina Radrizzani, Luigi Naldini

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Host-dependent resistance of Group A Streptococcus to sulfamethoxazole mediated by a horizontally-acquired reduced folate transporter by M. Kalindu D. Rodrigo, Aarti Saiganesh, Andrew J. Hayes, Alisha M. Wilson, Jack Anstey, Janessa L. Pickering, Jua Iwasaki, Jessica Hillas, Scott Winslow, Tabitha Woodman, Philipp Nitschke, Jake A. Lacey, Karen J. Breese, Mark P. G. van der Linden, Philip M. Giffard, Steven Y. C. Tong, Nicola Gray, Keith A. Stubbs, Jonathan R. Carapetis, Asha C. Bowen, Mark R. Davies, Timothy C. Barnett

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The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy by Luigi Naldini, Maria Pia Cicalese, Maria Ester Bernardo, Bernhard Gentner, Michela Gabaldo, Giuliana Ferrari, Alessandro Aiuti

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S106: LONG-TERM FOLLOW-UP OF BETA-THALASSEMIA PATIENTS TREATED WITH HEMATOPOIETIC STEM CELL GENE THERAPY by S Marktel, S Scaramuzza, F Giglio, M Cicalese, M Lidonnici, C Rossi, V Calbi, N Masera, E D’Angelo, N Mirra, R Origa, I Tartaglione, S Perrotta, G Viarengo, L Santoleri, R Milani, S Gattillo, A Calabria, E Montini, G Graziadei, L Naldini, M Cappellini, A Aiuti, F Ciceri, G Ferrari

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Evaluating COVID-19-Related Disruptions to Effective Malaria Case Management in 2020–2021 and Its Potential Effects on Malaria Burden in Sub-Saharan Africa by Paulina A. Dzianach, Susan F. Rumisha, Jailos Lubinda, Adam Saddler, Mauricio van den Berg, Yalemzewod A. Gelaw, Joseph R. Harris, Annie J. Browne, Francesca Sanna, Jennifer A. Rozier, Beatriz Galatas, Laura F. Anderson, Camilo A. Vargas-Ruiz, Ewan Cameron, Peter W. Gething, Daniel J. Weiss

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