Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID)

por Booth, Claire, Gaspar, H Bobby
Publicado 2009

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

por Montiel-Equihua, Claudia A, Thrasher, Adrian J, Gaspar, H Bobby
Publicado 2009

Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease

por Panchal, Neelam, Houghton, Ben, Diez, Begona, Ghosh, Sujal, Ricciardelli, Ida, Thrasher, Adrian J., Gaspar, H. Bobby, Booth, Claire
Publicado 2018

Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience

por Kreins, Alexandra Y., Velasco, Helena F., Cheong, Kai-Ning, Rao, Kanchan, Veys, Paul, Worth, Austen, Gaspar, H. Bobby, Booth, Claire
Publicado 2021

Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency

por Topal, Joseph, Panchal, Neelam, Barroeta, Amairelys, Roppelt, Anna, Mudde, Annelotte, Gaspar, H. Bobby, Thrasher, Adrian J., Houghton, Benjamin C., Booth, Claire
Publicado 2022

Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cel...

por Zhang, Fang, Thornhill, Susannah I., Howe, Steven J., Ulaganathan, Meera, Schambach, Axel, Sinclair, Joanna, Kinnon, Christine, Gaspar, H. Bobby, Antoniou, Michael, Thrasher, Adrian J.
Publicado 2007

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

por Poletti, Valentina, Charrier, Sabine, Corre, Guillaume, Gjata, Bernard, Vignaud, Alban, Zhang, Fang, Rothe, Michael, Schambach, Axel, Gaspar, H. Bobby, Thrasher, Adrian J., Mavilio, Fulvio
Publicado 2018

Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy

por Moretti, Federico A., Giardino, Giuliana, Attenborough, Teresa C. H., Gkazi, Athina Soragia, Margetts, Ben K., la Marca, Giancarlo, Fairbanks, Lynette, Crompton, Tessa, Gaspar, H. Bobby
Publicado 2021

Author Correction: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy

por Moretti, Federico A., Giardino, Giuliana, Attenborough, Teresa C. H., Gkazi, Athina Soragia, Margetts, Ben K., la Marca, Giancarlo, Fairbanks, Lynette, Crompton, Tessa, Gaspar, H. Bobby
Publicado 2021

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

por Izotova, Natalia, Rivat, Christine, Baricordi, Cristina, Blanco, Elena, Pellin, Danilo, Watt, Eleanor, Gkazi, Athina S., Adams, Stuart, Gilmour, Kimberly, Bayford, Jinhua, Booth, Claire, Gaspar, H. Bobby, Thrasher, Adrian J., Biasco, Luca
Publicado 2021

T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations

por Ghosh, Sujal, Carmo, Marlene, Calero-Garcia, Miguel, Ricciardelli, Ida, Bustamante Ogando, Juan Carlos, Blundell, Michael P., Schambach, Axel, Ashton-Rickardt, Philip G., Booth, Claire, Ehl, Stephan, Lehmberg, Kai, Thrasher, Adrian J., Gaspar, H. Bobby
Publicado 2018

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

por Ellison, Stuart M., Liao, Aiyin, Wood, Shaun, Taylor, Jessica, Youshani, Amir Saam, Rowlston, Sam, Parker, Helen, Armant, Myriam, Biffi, Alessandra, Chan, Lucas, Farzaneh, Farzin, Wynn, Rob, Jones, Simon A., Heal, Paul, Gaspar, H. Bobby, Bigger, Brian W.
Publicado 2019

Perforin Gene Transfer Into Hematopoietic Stem Cells Improves Immune Dysregulation in Murine Models of Perforin Deficiency

por Carmo, Marlene, Risma, Kimberly A, Arumugam, Paritha, Tiwari, Swati, Hontz, Adrianne E, Montiel-Equihua, Claudia A, Alonso-Ferrero, Maria E, Blundell, Michael P, Schambach, Axel, Baum, Christopher, Malik, Punam, Thrasher, Adrian J, Jordan, Michael B, Gaspar, H Bobby
Publicado 2015

Molecular Pathogenesis of EBV Susceptibility in XLP as Revealed by Analysis of Female Carriers with Heterozygous Expression of SAP

por Palendira, Umaimainthan, Low, Carol, Chan, Anna, Hislop, Andrew D., Ho, Edwin, Phan, Tri Giang, Deenick, Elissa, Cook, Matthew C., Riminton, D. Sean, Choo, Sharon, Loh, Richard, Alvaro, Frank, Booth, Claire, Gaspar, H. Bobby, Moretta, Alessandro, Khanna, Rajiv, Rickinson, Alan B., Tangye, Stuart G.
Publicado 2011

Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ()

por Burns, Siobhan O., Plagnol, Vincent, Gutierrez, Beatriz Morillo, Al Zahrani, Daifulah, Curtis, James, Gaspar, Miguel, Hassan, Amel, Jones, Alison M., Malone, Marian, Rampling, Dyanne, McLatchie, Alex, Doffinger, Rainer, Gilmour, Kimberly C., Henriquez, Frances, Thrasher, Adrian J., Gaspar, H. Bobby, Nejentsev, Sergey
Publicado 2014

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

por Piras, Giuseppa, Montiel-Equihua, Claudia, Chan, Yee-Ka Agnes, Wantuch, Slawomir, Stuckey, Daniel, Burke, Derek, Prunty, Helen, Phadke, Rahul, Chambers, Darren, Partida-Gaytan, Armando, Leon-Rico, Diego, Panchal, Neelam, Whitmore, Kathryn, Calero, Miguel, Benedetti, Sara, Santilli, Giorgia, Thrasher, Adrian J., Gaspar, H. Bobby
Publicado 2020

Primary Immunodeficiency Diseases: An Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency

por Al-Herz, Waleed, Bousfiha, Aziz, Casanova, Jean-Laurent, Chatila, Talal, Conley, Mary Ellen, Cunningham-Rundles, Charlotte, Etzioni, Amos, Franco, Jose Luis, Gaspar, H. Bobby, Holland, Steven M., Klein, Christoph, Nonoyama, Shigeaki, Ochs, Hans D., Oksenhendler, Erik, Picard, Capucine, Puck, Jennifer M., Sullivan, Kate, Tang, Mimi L. K.
Publicado 2014

Corrigendum: Primary Immunodeficiency Diseases: An Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficienc...

por Al-Herz, Waleed, Bousfiha, Aziz, Casanova, Jean-Laurent, Chatila, Talal, Conley, Mary Ellen, Cunningham-Rundles, Charlotte, Etzioni, Amos, Franco, Jose Luis, Gaspar, H. Bobby, Holland, Steven M., Klein, Christoph, Nonoyama, Shigeaki, Ochs, Hans D., Oksenhendler, Erik, Picard, Capucine, Puck, Jennifer M., Sullivan, Kate, Tang, Mimi L. K.
Publicado 2014

Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood

por Day, James W., Elfeky, Reem, Nicholson, Bethany, Goodman, Rupert, Pearce, Rachel, Fox, Thomas A., Worth, Austen, Booth, Claire, Veys, Paul, Carpenter, Ben, Hough, Rachael, Gaspar, H. Bobby, Titman, Penny, Ridout, Deborah, Workman, Sarita, Hernandes, Fernando, Sandford, Kit, Laurence, Arian, Campbell, Mari, Burns, Siobhan O., Morris, Emma C.
Publicado 2022

Primary Immunodeficiency Diseases: an Update on the Classification from the International Union of Immunological Societies Expert Committee for Primary Immunodeficiency 2015

por Picard, Capucine, Al-Herz, Waleed, Bousfiha, Aziz, Casanova, Jean-Laurent, Chatila, Talal, Conley, Mary Ellen, Cunningham-Rundles, Charlotte, Etzioni, Amos, Holland, Steven M., Klein, Christoph, Nonoyama, Shigeaki, Ochs, Hans D., Oksenhendler, Eric, Puck, Jennifer M., Sullivan, Kathleen E., Tang, Mimi L K., Franco, Jose Luis, Gaspar, H. Bobby
Publicado 2015