[HTML][HTML] Efficacy and safety of anti-CD45–saporin as conditioning agent for RAG deficiency

MC Castiello, M Bosticardo, N Sacchetti… - Journal of Allergy and …, 2021 - Elsevier
Background Mutations in the recombinase-activating genes cause severe
immunodeficiency, with a spectrum of phenotypes ranging from severe combined
immunodeficiency to immune dysregulation. Hematopoietic stem cell transplantation is the
only curative option, but a high risk of graft failure and poor immune reconstitution have
been observed in the absence of myeloablation. Objectives Our aim was to improve
multilineage engraftment; we tested nongenotoxic conditioning with anti-CD45 mAbs …