Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID
Laura García-Pérez, 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 C. Lankester, Frank J. T. Staal, Karin Pike‐Overzet
Abstract
cells transduced with our clinical RAG1 vector and transplanted into NSG mice led to improved human B and T cell development. Considering this efficacy outcome, together with favorable safety data, these results substantiate the need for a clinical trial for RAG1-SCID.
Topics & Concepts
Genetic enhancementGeneRecombinaseBiologyComputational biologyMedicineGeneticsCancer researchRecombinationVirus-based gene therapy researchCAR-T cell therapy researchCRISPR and Genetic Engineering