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

od Booth, Claire, Gaspar, H Bobby
Izdano 2009

Update on clinical gene therapy in childhood

od Qasim, Waseem, Gaspar, H Bobby, Thrasher, Adrian J
Izdano 2007

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

od Gaspar, H Bobby, Swift, Sue, Thrasher, Adrian J
Izdano 2013

Gene Therapy for Primary Immunodeficiencies

od Rivat, Christine, Santilli, Giorgia, Gaspar, H. Bobby, Thrasher, Adrian J.
Izdano 2012

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

od Montiel-Equihua, Claudia A, Thrasher, Adrian J, Gaspar, H Bobby
Izdano 2009

How I treat ADA deficiency

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

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

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

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

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

Sleeping Beauty Transposition From Nonintegrating Lentivirus

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

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

od Kohn, Donald B., Hershfield, Michael S., Puck, Jennifer M., Aiuti, Alessandro, Blincoe, Annaliesse, Gaspar, H. Bobby, Notarangelo, Luigi D., Grunebaum, Eyal
Izdano u 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

od Debnath, Shubhranshu, Jaako, Pekka, Siva, Kavitha, Rothe, Michael, Chen, Jun, Dahl, Maria, Gaspar, H. Bobby, Flygare, Johan, Schambach, Axel, Karlsson, Stefan
Izdano u 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...

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

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

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

Busulfan Pharmacokinetics in ADA SCID Gene Therapy

od 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.
Izdano u Biol Blood Marrow Transplant (2020)

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

od 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
Izdano 2007

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

od 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
Izdano u Mol Ther (2008)

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

od 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
Izdano u Nat Commun (2021)

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

od 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
Izdano u 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

od 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.
Izdano u Mol Ther Methods Clin Dev (2019)

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

od 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
Izdano u Mol Ther (2015)