A Practical Guide to Implementing Population Newborn Screening (NBS) for Severe Combined Immunodeficiency (SCID)

by H. Bobby Gaspar
Published 2017-11-01

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

by Claire Booth, H Bobby Gaspar
Published 2009-06-01

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

by Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby Gaspar
Published 2009-12-01

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

by Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio
Published 2018-06-01

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

by Natalia Izotova, Christine Rivat, Cristina Baricordi, Elena Blanco, Danilo Pellin, Eleanor Watt, Athina S. Gkazi, Stuart Adams, Kimberly Gilmour, Jinhua Bayford, Claire Booth, H. Bobby Gaspar, Adrian J. Thrasher, Luca Biasco
Published 2021-03-01

Autologous Transplantation of Amniotic Fluid-Derived Mesenchymal Stem Cells into Sheep Fetuses

by S. W. Steven Shaw, Sveva Bollini, Khalil Abi Nader, Annalisa Gastaldello, Vedanta Mehta, Elisa Filppi, Mara Cananzi, H. Bobby Gaspar, Waseem Qasim, Paolo De Coppi M.D., Ph.D., Anna L. David
Published 2016-03-01

Autologous Transplantation of Amniotic Fluid-Derived Mesenchymal Stem Cells into Sheep Fetuses

by S. W. Steven Shaw, Sveva Bollini, Khalil Abi Nader, Annalisa Gastadello, Vedanta Mehta, Elisa Filppi, Mara Cananzi, H. Bobby Gaspar, Waseem Qasim, Paolo De Coppi M.D., Ph.D., Anna L. David M.D., Ph.D.
Published 2011-08-01

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

by Stuart M. Ellison, Aiyin Liao, Shaun Wood, Jessica Taylor, Amir Saam Youshani, Sam Rowlston, Helen Parker, Myriam Armant, Alessandra Biffi, Lucas Chan, Farzin Farzaneh, Rob Wynn, Simon A. Jones, Paul Heal, H. Bobby Gaspar, Brian W. Bigger
Published 2019-06-01

Molecular pathogenesis of EBV susceptibility in XLP as revealed by analysis of female carriers with heterozygous expression of SAP.

by Umaimainthan Palendira, Carol Low, Anna Chan, Andrew D Hislop, Edwin Ho, Tri Giang Phan, Elissa Deenick, Matthew C Cook, D Sean Riminton, Sharon Choo, Richard Loh, Frank Alvaro, Claire Booth, H Bobby Gaspar, Alessandro Moretta, Rajiv Khanna, Alan B Rickinson, Stuart G Tangye
Published 2011-11-01

Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

by Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel Stuckey, Derek Burke, Helen Prunty, Rahul Phadke, Darren Chambers, Armando Partida-Gaytan, Diego Leon-Rico, Neelam Panchal, Kathryn Whitmore, Miguel Calero, Sara Benedetti, Giorgia Santilli, Adrian J. Thrasher, H. Bobby Gaspar
Published 2020-09-01

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

by Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A. Vloemans, Martijn Cordes, Axel Schambach, Michael Rothe, Dagmar Berghuis, Chantal Lagresle-Peyrou, Marina Cavazzana, Fang Zhang, Adrian J. Thrasher, Daniela Salvatori, Pauline Meij, Anna Villa, Jacques J.M. Van Dongen, Jaap-Jan Zwaginga, Mirjam van der Burg, H. Bobby Gaspar, Arjan Lankester, Frank J.T. Staal, Karin Pike-Overzet
Published 2020-06-01