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 2009

Identification of a small molecule for enhancing lentiviral transduction of T cells

por Malach, Paulina, Kay, Charlotte, Tinworth, Chris, Patel, Florence, Joosse, Bryan, Wade, Jennifer, Rosa do Carmo, Marlene, Donovan, Brian, Brugman, Martijn, Montiel-Equihua, Claudia, Francis, Natalie
Publicado 2023

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 2015

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

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