Adenosine Deaminase Deficiency – More Than Just an Immunodeficiency

por Whitmore, Kathryn V., Gaspar, Hubert B.
Publicado 2016

Universal Newborn Screening for Severe Combined Immunodeficiency (SCID)

por van der Burg, Mirjam, Mahlaoui, Nizar, Gaspar, Hubert Bobby, Pai, Sung-Yun
Publicado 2019

Primary Immune Deficiencies – Principles of Care

por Chapel, Helen, Prevot, Johan, Gaspar, Hubert Bobby, Español, Teresa, Bonilla, Francisco A., Solis, Leire, Drabwell, Josina
Publicado 2014

Production and First-in-Man Use of T Cells Engineered to Express a HSVTK-CD34 Sort-Suicide Gene

por Zhan, Hong, Gilmour, Kimberly, Chan, Lucas, Farzaneh, Farzin, McNicol, Anne Marie, Xu, Jin-Hua, Adams, Stuart, Fehse, Boris, Veys, Paul, Thrasher, Adrian, Gaspar, Hubert, Qasim, Waseem
Publicado 2013

Lentiviral gene therapy rescues p47(phox) chronic granulomatous disease and the ability to fight Salmonella infection in mice

por Schejtman, Andrea, Aragão-Filho, Walmir Cutrim, Clare, Simon, Zinicola, Marta, Weisser, Maren, Burns, Siobhan O., Booth, Claire, Gaspar, Hubert B., Thomas, David C., Condino-Neto, Antonio, Thrasher, Adrian J., Santilli, Giorgia
Publicado 2020

Host natural killer immunity is a key indicator of permissiveness for donor cell engraftment in patients with severe combined immunodeficiency()

por Hassan, Amel, Lee, Pamela, Maggina, Paraskevi, Xu, Jin Hua, Moreira, Diana, Slatter, Mary, Nademi, Zohreh, Worth, Austen, Adams, Stuart, Jones, Alison, Cale, Catherine, Allwood, Zoe, Rao, Kanchan, Chiesa, Robert, Amrolia, Persis, Gaspar, Hubert, Davies, E. Graham, Veys, Paul, Gennery, Andrew, Qasim, Waseem
Publicado 2014

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47(phox)-Deficient Chronic Granulomatous Disease

por Schejtman, Andrea, Vetharoy, Winston, Choi, Uimook, Rivat, Christine, Theobald, Narda, Piras, Giuseppa, Leon-Rico, Diego, Buckland, Karen, Armenteros-Monterroso, Elena, Benedetti, Sara, Ashworth, Michael T., Rothe, Michael, Schambach, Axel, Gaspar, Hubert Bobby, Kang, Elizabeth M., Malech, Harry L., Thrasher, Adrian J., Santilli, Giorgia
Publicado 2021

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

por Schwarzer, Adrian, Talbot, Steven R., Selich, Anton, Morgan, Michael, Schott, Juliane W., Dittrich-Breiholz, Oliver, Bastone, Antonella L., Weigel, Bettina, Ha, Teng Cheong, Dziadek, Violetta, Gijsbers, Rik, Thrasher, Adrian J., Staal, Frank J.T., Gaspar, Hubert B., Modlich, Ute, Schambach, Axel, Rothe, Michael
Publicado 2021