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

Por Booth, Claire, Gaspar, H Bobby
Publicado em 2009

Update on clinical gene therapy in childhood

Por Qasim, Waseem, Gaspar, H Bobby, Thrasher, Adrian J
Publicado em 2007

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

Por Gaspar, H Bobby, Swift, Sue, Thrasher, Adrian J
Publicado em 2013

Gene Therapy for Primary Immunodeficiencies

Por Rivat, Christine, Santilli, Giorgia, Gaspar, H. Bobby, Thrasher, Adrian J.
Publicado em 2012

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 em 2009

How I treat ADA deficiency

Por Gaspar, H. Bobby, Aiuti, Alessandro, Porta, Fulvio, Candotti, Fabio, Hershfield, Michael S., Notarangelo, Luigi D.
Publicado em 2009

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

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

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 no J Allergy Clin Immunol (2018)

Sleeping Beauty Transposition From Nonintegrating Lentivirus

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

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

Por Kohn, Donald B., Hershfield, Michael S., Puck, Jennifer M., Aiuti, Alessandro, Blincoe, Annaliesse, Gaspar, H. Bobby, Notarangelo, Luigi D., Grunebaum, Eyal
Publicado no 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

Por Debnath, Shubhranshu, Jaako, Pekka, Siva, Kavitha, Rothe, Michael, Chen, Jun, Dahl, Maria, Gaspar, H. Bobby, Flygare, Johan, Schambach, Axel, Karlsson, Stefan
Publicado no 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...

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 em 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 no Mol Ther Methods Clin Dev (2018)

Busulfan Pharmacokinetics in ADA SCID Gene Therapy

Por 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.
Publicado no Biol Blood Marrow Transplant (2020)

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

Por 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
Publicado em 2007

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

Por 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
Publicado no Mol Ther (2008)

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 no Nat Commun (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 no 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

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 no Mol Ther Methods Clin Dev (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 no Mol Ther (2015)