Ogata J, Shimada Y, Ohashi T, Kobayashi H (2024) Usefulness of antibody-drug conjugate as preconditioning for hematopoietic stem cell-targeted gene therapy in wild-type and Fabry disease mouse models. Mol Genet Metab 142(3):108494. doi: 10.1016/j.ymgme.2024.108494 PMID: 38820907
Objective: To develop a saporin/antibody-based conditioning for hematopoietic stem cell-targeted gene therapy for use in models of Fabry Disease.
Summary: Fabry disease is characterized by deficient activity of α-galactosidase A (GLA). Consequently, globotriaosylceramide (Gb3) accumulates in various organs causing damage. Fabry Disease is treatable with hematopoietic stem cell therapy which requires strong conditioning, but that can often be associated with side effects. CD45-SAP is investigated as a conditioning agent for these stem cells.
Usage: CD45-SAP was used in hematopoietic stem cells in a mice model of Fabry Disease.