Efficacy and safety of anti-CD45-saporin as conditioning agent for RAG deficiency.

ABSTRACT:

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 conjugated with saporin CD45 (CD45-SAP).

Methods

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 (

SUBMITTER: Castiello MC 

PROVIDER: S-EPMC8322962 | biostudies-literature | 2021 Jan

REPOSITORIES: biostudies-literature