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

由 Booth, Claire, Gaspar, H Bobby
出版 2009

Update on clinical gene therapy in childhood

由 Qasim, Waseem, Gaspar, H Bobby, Thrasher, Adrian J
出版 2007

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

由 Gaspar, H Bobby, Swift, Sue, Thrasher, Adrian J
出版 2013

Gene Therapy for Primary Immunodeficiencies

由 Rivat, Christine, Santilli, Giorgia, Gaspar, H. Bobby, Thrasher, Adrian J.
出版 2012

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

由 Montiel-Equihua, Claudia A, Thrasher, Adrian J, Gaspar, H Bobby
出版 2009

How I treat ADA deficiency

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

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

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

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

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

Sleeping Beauty Transposition From Nonintegrating Lentivirus

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

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

由 Kohn, Donald B., Hershfield, Michael S., Puck, Jennifer M., Aiuti, Alessandro, Blincoe, Annaliesse, Gaspar, H. Bobby, Notarangelo, Luigi D., Grunebaum, Eyal
發表在 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

由 Debnath, Shubhranshu, Jaako, Pekka, Siva, Kavitha, Rothe, Michael, Chen, Jun, Dahl, Maria, Gaspar, H. Bobby, Flygare, Johan, Schambach, Axel, Karlsson, Stefan
發表在 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...

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

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

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

Busulfan Pharmacokinetics in ADA SCID Gene Therapy

由 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.
發表在 Biol Blood Marrow Transplant (2020)

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

由 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
出版 2007

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

由 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
發表在 Mol Ther (2008)

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

由 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
發表在 Nat Commun (2021)

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

由 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
發表在 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

由 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.
發表在 Mol Ther Methods Clin Dev (2019)

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

由 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
發表在 Mol Ther (2015)