{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Castiello MC"],"funding":["Intramural NIH HHS","Ministero dell’Istruzione, dell’Università e della Ricerca","Division of Intramural Research, National Institute of Allergy and Infectious Diseases"],"pagination":["309-320.e6"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8322962"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["147(1)"],"pubmed_abstract":["<h4>Background</h4>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.<h4>Objectives</h4>Our aim was to improve multilineage engraftment; we tested nongenotoxic conditioning with anti-CD45 mAbs conjugated with saporin CD45 (CD45-SAP).<h4>Methods</h4>Rag1-KO and Rag1-F971L mice, which represent models of severe combined immune deficiency and combined immune deficiency with immune dysregulation, respectively, were conditioned with CD45-SAP, CD45-SAP plus 2 Gy of total body irradiation ("],"journal":["The Journal of allergy and clinical immunology"],"pubmed_title":["Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency."],"pmcid":["PMC8322962"],"funding_grant_id":["ZIA AI001222"],"pubmed_authors":["Castiello MC","Villa A","Bosticardo M","Fontana E","Corsino C","Notarangelo LD","Sereni L","Yamazaki Y","Sacchetti N","Uva P","Palchaudhuri R","Calzoni E","Scadden DT","Bortolomai I","Draghici E","Yu HH"],"additional_accession":[]},"is_claimable":false,"name":"Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency.","description":"<h4>Background</h4>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.<h4>Objectives</h4>Our aim was to improve multilineage engraftment; we tested nongenotoxic conditioning with anti-CD45 mAbs conjugated with saporin CD45 (CD45-SAP).<h4>Methods</h4>Rag1-KO and Rag1-F971L mice, which represent models of severe combined immune deficiency and combined immune deficiency with immune dysregulation, respectively, were conditioned with CD45-SAP, CD45-SAP plus 2 Gy of total body irradiation (","dates":{"release":"2021-01-01T00:00:00Z","publication":"2021 Jan","modification":"2026-05-09T17:15:27.849Z","creation":"2022-02-11T00:53:19.1Z"},"accession":"S-EPMC8322962","cross_references":{"pubmed":["32387109"],"doi":["10.1016/j.jaci.2020.04.033"]}}