Not logged in Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency Booth, Claire; Masiuk, Katelyn; Vazouras, Konstantinos; Fernandes, Augustine; Xu-Bayford, Jinhua; Campo Fernandez, Beatriz; Roy, Sohini; Curio-Penny, Beatrice; Arnold, Jordyn; Terrazas, Dayna; Reid, Jack; Gilmour, Kimberly C.; Adams, Stuart; Alvarez Mediavilla, Elena; Mhaldien, Lana; O'Toole, Grainne; Ahmed, Rima; Garabedian, Elizabeth; Malech, Harry; De Ravin, Suk See; Moore, Theodore B.; De Oliveira, Satiro; Pellin, Danilo; Lin, Tsai-Yu; Dang, Thao Thi; Cornetta, Kenneth; Hershfield, Michael S.; Hara, Havinder; Thrasher, Adrian J.; Gaspar, H. Bobby; Kohn, Donald B. Share Save
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Dominant negative variants in ITPR3 impair T cell Ca2+ dynamics causing combined immunodeficiency Blanco, Elena; Camps, Carme; Bahal, Sameer; Kerai, Mohit D.; Ferla, Matteo P.; Rochussen, Adam M.; Handel, Adam E.; Golwala, Zainab M.; Spiridou Goncalves, Helena; Kricke, Susanne; Klein, Fabian; Zhang, Fang; Zinghirino, Federica; Evans, Grace; Keane, Thomas M.; Lizot, Sabrina; Kusters, Maaike A. A.; Iro, Mildred A.; Patel, Sanjay V.; Morris, Emma C.; Burns, Siobhan O.; Radcliffe, Ruth; Vasudevan, Pradeep; Price, Arthur; Gillham, Olivia; Valdebenito, Gabriel E.; Stewart, Grant S.; Worth, Austen; Adams, Stuart P.; Duchen, Michael; Andre, Isabelle; Adams, David J.; Santili, Giorgia; Gilmour, Kimberly C.; Hollaender, Georg A.; Davies, E. Graham; Taylor, Jenny C.; Griffiths, Gillian M.; Thrasher, Adrian J.; Dhalla, Fatima; Kreins, Alexandra Y. Share Save
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial Kwiatkowski, Janet L.; Walters, Mark C.; Hongeng, Suradej; Yannaki, Evangelia; Kulozik, Andreas E.; Kunz, Joachim B.; Sauer, Martin G.; Thrasher, Adrian J.; Thuret, Isabelle; Lal, Ashutosh; Tao, Ge; Ali, Shamshad; Thakar, Himal L.; Elliot, Heidi; Lodaya, Ankit; Lee, Ji; Colvin, Richard A.; Locatelli, Franco; Thompson, Alexis A. Share Save
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy Eichler, Florian; Duncan, Christine N.; Musolino, Patricia L.; Lund, Troy C.; Gupta, Ashish O.; De Oliveira, Satiro; Thrasher, Adrian J.; Aubourg, Patrick; Kuehl, Joern-Sven; Loes, Daniel J.; Amartino, Hernan; Smith, Nicholas; Fernandes, Juliana Folloni; Sevin, Caroline; Sankar, Raman; Hussain, Shaun A.; Gissen, Paul; Dalle, Jean-Hugues; Platzbecker, Uwe; Downey, Gerald F.; McNeil, Elizabeth; Demopoulos, Laura; Dietz, Andrew C.; Thakar, Himal L.; Orchard, Paul J.; Williams, David A. Share Save
Valproic Acid Confers Functional Pluripotency to Human Amniotic Fluid Stem Cells in a Transgene-free Approach Moschidou, Dafni; Mukherjee, Sayandip; Blundell, Michael P.; Drews, Katharina; Jones, Gemma N.; Abdulrazzak, Hassan; Nowakowska, Beata; Phoolchund, Anju; Lay, Kenneth; Ramasamy, T. Selvee; Cananzi, Mara; Nettersheim, Daniel; Sullivan, Mark; Frost, Jennifer; Moore, Gudrun; Vermeesch, Joris R.; Fisk, Nicholas M.; Thrasher, Adrian J.; Atala, Anthony; Adjaye, James; Schorle, Hubert; De Coppi, Paolo; V. Guillot, Pascale Share Save
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia Bahal, Sameer; Zinicola, Marta; Moula, Shefta E.; Whittaker, Thomas E.; Schejtman, Andrea; Naseem, Asma; Blanco, Elena; Vetharoy, Winston; Hu, Yi-Ting; Rai, Rajeev; Gomez-Castaneda, Eduardo; Cunha-Santos, Catarina; Burns, Siobhan O.; Morris, Emma C.; Booth, Claire; Turchiano, Giandomenico; Cavazza, Alessia; Thrasher, Adrian J.; Santilli, Giorgia Share Save
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant Yeung, Jenny; Liao, Aiyin; Shaw, Matthew; Silva, Soraia; Vetharoy, Winston; Rico, Diego Leon; Kirby, Ian; Zammarchi, Francesca; Havenith, Karin; de Haan, Lolke; van Berkel, Patrick H.; Sebire, Neil; Ogunbiyi, Olumide K.; Booth, Claire; Gaspar, H. Bobby; Thrasher, Adrian J.; Chester, Kerry A.; Amrolia, Persis J. Share Save
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV Vaidyanathan, Sriram; Kerschner, Jenny L.; Paranjapye, Alekh; Sinha, Vrishti; Lin, Brian; Bedrosian, Tracy A.; Thrasher, Adrian J.; Turchiano, Giandomenico; Harris, Ann; Porteus, Matthew H. Share Save
AAV capsid bioengineering in primary human retina models Westhaus, Adrian; Eamegdool, Steven S.; Fernando, Milan; Fuller-Carter, Paula; Brunet, Alicia A.; Miller, Annie L.; Rashwan, Rabab; Knight, Maddison; Daniszewski, Maciej; Lidgerwood, Grace E.; Pebay, Alice; Hewitt, Alex; Santilli, Giorgia; Thrasher, Adrian J.; Carvalho, Livia S.; Gonzalez-Cordero, Anai; Jamieson, Robyn V.; Lisowski, Leszek Share Save
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials Cornetta, Kenneth; Yao, Jing; House, Kimberley; Duffy, Lisa; Adusumilli, Prasad S.; Beyer, Rachel; Booth, Claire; Brenner, Malcolm; Curran, Kevin; Grilley, Bambi; Heslop, Helen; Hinrichs, Christian S.; Kaplan, Rosandra N.; Kiem, Hans-Peter; Kochenderfer, James; Kohn, Donald B.; Mailankody, Sham; Norberg, Scott M.; O'Cearbhaill, Roisin E.; Pappas, Jennifer; Park, Jae; Ramos, Carlos; Ribas, Antonio; Riviere, Isabelle; Rosenberg, Steven A.; Sauter, Craig; Shah, Nirali N.; Slovin, Susan F.; Thrasher, Adrian; Williams, David A.; Lin, Tsai-Yu Share Save
Access to gene therapy for rare diseases when commercialization is not fit for purpose Fox, Thomas; Bueren, Juan; Candotti, Fabio; Fischer, Alain; Aiuti, Alessandro; Lankester, Arjan; AGORA Initiative, Michael; Albert, Michael; Bernardo, Maria Ester; Cavazzana, Marina; Ehl, Stephan; Neven, Benedicte; Prevot, Johan; Schambach, Axel; Sevilla, Julian; Thrasher, Adrian; Wynn, Rob; Booth, Claire Share Save
Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy Sobrino, Steicy; Magnani, Alessandra; Semeraro, Michaela; Martignetti, Loredana; Cortal, Akira; Denis, Adeline; Couzin, Chloe; Picard, Capucine; Bustamante, Jacinta; Magrin, Elisa; Joseph, Laure; Roudaut, Cecile; Gabrion, Aurelie; Soheili, Tayebeh; Cordier, Corinne; Lortholary, Olivier; Lefrere, Francois; Rieux-Laucat, Frederic; Casanova, Jean-Laurent; Bodard, Sylvain; Boddaert, Nathalie; Thrasher, Adrian J.; Touzot, Fabien; Taque, Sophie; Suarez, Felipe; Marcais, Ambroise; Guilloux, Agathe; Lagresle-Peyrou, Chantal; Galy, Anne; Rausell, Antonio; Blanche, Stephane; Cavazzana, Marina; Six, Emmanuelle Share Save
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Partial human Janus kinase 1 deficiency predominantly impairs responses to interferon gamma and intracellular control of mycobacteria Daza-Cajigal, Vanessa; Albuquerque, Adriana S.; Young, Dan F.; Ciancanelli, Michael J.; Moulding, Dale; Angulo, Ivan; Jeanne-Julien, Valentine; Rosain, Jeremie; Minskaia, Ekaterina; Casanova, Jean-Laurent; Boisson-Dupuis, Stephanie; Bustamante, Jacinta; Randall, Richard E.; McHugh, Timothy D.; Thrasher, Adrian J.; Burns, Siobhan O. Share Save
Immunodeficiency, autoimmunity, and increased risk of B cell malignancy in humans with TRAF3 mutations Rae, William; Sowerby, John M.; Verhoeven, Dorit; Youssef, Mariam; Kotagiri, Prasanti; Savinykh, Natalia; Coomber, Eve L.; Boneparth, Alexis; Chan, Angela; Gong, Chun; Jansen, Machiel H.; du Long, Romy; Santilli, Giorgia; Simeoni, Ilenia; Stephens, Jonathan; Wu, Kejia; Zinicola, Marta; Allen, Hana Lango; Baxendale, Helen; Kumararatne, Dinakantha; Gkrania-Klotsas, Effrossyni; Mendoza, Selma C. Scheffler; Yamazaki-Nakashimada, Marco Antonio; Ruiz, Laura Berron; Rojas-Maruri, Cesar Mauricio; Reyes, Saul O. Lugo; Lyons, Paul A.; Williams, Anthony P.; Hodson, Daniel J.; Bishop, Gail A.; Thrasher, Adrian J.; Thomas, David C.; Murphy, Michael P.; Vyse, Timothy J.; Milner, Joshua D.; Kuijpers, Taco W.; Smith, Kenneth G. C. Share Save
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome (Aug, 10.1038/s41591-021-01641-x, 2022) Magnani, A.; Semeraro, M.; Adam, F.; Booth, C.; Dupre, L.; Morris, E. C.; Gabrion, A.; Roudaut, C.; Borgel, D.; Toubert, A.; Clave, E.; Abdo, C.; Gorochov, G.; Petermann, R.; Guiot, M.; Miyara, M.; Moshous, D.; Magrin, E.; Denis, A.; Suarez, F.; Lagresle, C.; Roche, A. M.; Everett, J.; Trinquand, A.; Guisset, M.; Bayford, J. Xu; Hacein-Bey-Abina, S.; Kauskot, A.; Elfeky, R.; Rivat, C.; Abbas, S.; Gaspar, H. B.; Macintyre, E.; Picard, C.; Bushman, F. D.; Galy, A.; Fischer, A.; Six, E.; Thrasher, A. J.; Cavazzana, M. Share Save
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