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

gan Booth, Claire, Gaspar, H Bobby
Cyhoeddwyd 2009

Update on clinical gene therapy in childhood

gan Qasim, Waseem, Gaspar, H Bobby, Thrasher, Adrian J
Cyhoeddwyd 2007

“Special Exemptions”: Should They Be Put on Trial?

gan Gaspar, H Bobby, Swift, Sue, Thrasher, Adrian J
Cyhoeddwyd 2013

Gene Therapy for Primary Immunodeficiencies

gan Rivat, Christine, Santilli, Giorgia, Gaspar, H. Bobby, Thrasher, Adrian J.
Cyhoeddwyd 2012

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

gan Montiel-Equihua, Claudia A, Thrasher, Adrian J, Gaspar, H Bobby
Cyhoeddwyd 2009

How I treat ADA deficiency

gan Gaspar, H. Bobby, Aiuti, Alessandro, Porta, Fulvio, Candotti, Fabio, Hershfield, Michael S., Notarangelo, Luigi D.
Cyhoeddwyd 2009

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

gan Rivat, Christine, Booth, Claire, Alonso-Ferrero, Maria, Blundell, Michael, Sebire, Neil J., Thrasher, Adrian J., Gaspar, H. Bobby
Cyhoeddwyd 2013

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

gan Panchal, Neelam, Houghton, Ben, Diez, Begona, Ghosh, Sujal, Ricciardelli, Ida, Thrasher, Adrian J., Gaspar, H. Bobby, Booth, Claire
Cyhoeddwyd yn J Allergy Clin Immunol (2018)

Sleeping Beauty Transposition From Nonintegrating Lentivirus

gan Vink, Conrad A, Gaspar, H Bobby, Gabriel, Richard, Schmidt, Manfred, McIvor, R Scott, Thrasher, Adrian J, Qasim, Waseem
Cyhoeddwyd 2009

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

gan Kohn, Donald B., Hershfield, Michael S., Puck, Jennifer M., Aiuti, Alessandro, Blincoe, Annaliesse, Gaspar, H. Bobby, Notarangelo, Luigi D., Grunebaum, Eyal
Cyhoeddwyd yn J Allergy Clin Immunol (2018)

Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia

gan Debnath, Shubhranshu, Jaako, Pekka, Siva, Kavitha, Rothe, Michael, Chen, Jun, Dahl, Maria, Gaspar, H. Bobby, Flygare, Johan, Schambach, Axel, Karlsson, Stefan
Cyhoeddwyd yn Mol Ther (2017)

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

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

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

gan Poletti, Valentina, Charrier, Sabine, Corre, Guillaume, Gjata, Bernard, Vignaud, Alban, Zhang, Fang, Rothe, Michael, Schambach, Axel, Gaspar, H. Bobby, Thrasher, Adrian J., Mavilio, Fulvio
Cyhoeddwyd yn Mol Ther Methods Clin Dev (2018)

Busulfan Pharmacokinetics in ADA SCID Gene Therapy

gan Bradford, Kathryn L., Liu, Siyu, Krajinovic, Maja, Ansari, Marc, Garabedian, Elizabeth, Tse, John, Wang, Xiaoyan, Shaw, Kit L., Gaspar, H. Bobby, Candotti, Fabio, Kohn, Donald B.
Cyhoeddwyd yn Biol Blood Marrow Transplant (2020)

Reexamining the role of TACI coding variants in common variable immunodeficiency and selective IgA deficiency

gan Pan-Hammarström, Qiang, Salzer, Ulrich, Du, Likun, Björkander, Janne, Cunningham-Rundles, Charlotte, Nelson, David L, Bacchelli, Chiara, Gaspar, H Bobby, Offer, Steven, Behrens, Timothy W, Grimbacher, Bodo, Hammarström, Lennart
Cyhoeddwyd 2007

Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency

gan Thornhill, Susannah I, Schambach, Axel, Howe, Steven J, Ulaganathan, Meera, Grassman, Elke, Williams, David, Schiedlmeier, Bernhard, Sebire, Neil J, Gaspar, H Bobby, Kinnon, Christine, Baum, Christopher, Thrasher, Adrian J
Cyhoeddwyd yn Mol Ther (2008)

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

gan 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
Cyhoeddwyd yn Nat Commun (2021)

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

gan 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
Cyhoeddwyd yn J Allergy Clin Immunol (2018)

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

gan 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.
Cyhoeddwyd yn Mol Ther Methods Clin Dev (2019)

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

gan 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
Cyhoeddwyd yn Mol Ther (2015)