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

od Booth, Claire, Gaspar, H Bobby
Wydane 2009

Update on clinical gene therapy in childhood

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

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

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

Gene Therapy for Primary Immunodeficiencies

od Rivat, Christine, Santilli, Giorgia, Gaspar, H. Bobby, Thrasher, Adrian J.
Wydane 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
Wydane 2009

How I treat ADA deficiency

od Gaspar, H. Bobby, Aiuti, Alessandro, Porta, Fulvio, Candotti, Fabio, Hershfield, Michael S., Notarangelo, Luigi D.
Wydane 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
Wydane 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
Wydane w 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
Wydane 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
Wydane w 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
Wydane w 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.
Wydane 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
Wydane w 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.
Wydane w 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
Wydane 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
Wydane w 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
Wydane w 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
Wydane w 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.
Wydane w 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
Wydane w Mol Ther (2015)