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Effective Multi-lineage Engraftment in a Mouse Model of Fanconi Anemia Using Non-genotoxic Antibody-Based Conditioning

Meera Srikanthan, Olivier Humbert, Kevin G. Haworth, Christina Ironside, Yogendra Singh Rajawat, Bruce R. Blazar, Rahul Palchaudhuri, Anthony E. Boitano, M. Cooke, David T. Scadden, Hans‐Peter Kiem

2020Molecular Therapy — Methods & Clinical Development32 citationsDOIOpen Access PDF

Abstract

knockout mice were conditioned with either CD45-SAP or CD117-SAP prior to receiving whole marrow from a heterozygous healthy donor. Bone marrow and peripheral blood analysis revealed equivalent levels of donor engraftment, with minimal toxicity in ADC-treated groups as compared with cyclophosphamide-treated controls. Our findings suggest ADCs may be an effective conditioning strategy in stem cell transplantation not only for diseases where traditional chemotherapy is not tolerated, but also more broadly for the field of blood and marrow transplantation.

Topics & Concepts

Fanconi anemiaFANCABone marrowCancer researchImmunologyBone marrow failureDyskeratosis congenitaHaematopoiesisTransplantationCyclophosphamideMedicineStem cellBiologyChemotherapyInternal medicineBiochemistryGeneticsGeneDNADNA repairTelomereBlood disorders and treatmentsHematopoietic Stem Cell TransplantationErythrocyte Function and Pathophysiology