De Pontia G (2021) Exploring early therapeutic approaches in a mucopolysaccharidosis type I (MPSI) mouse model. Univ Milan-Bicocca Thesis.
Objective: Author applied hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT) treatments, individually or in combination, during the neonatal period, to evaluate the outcomes in a Mucopolysaccharidosis type I (MPS-I) mouse model.
Summary: Although the impact of the immune response on the outcome remains unknown and debated, authors demonstrated that the combination therapy permitted immune tolerance, avoiding anti-IDUA antibody development. Taken together, the data demonstrate that the combination of HSCT and ERT at a neonatal age may represent a beneficial therapeutic option for patients with MPS-I.
Usage: Using both CD45-SAP and/or CD117-SAP resulted in depletion capability similar to Total body irradiation (TBI) and stable unbiased high donor chimerism in adult immunocompetent mice, with architectural maintenance and reduced toxicity.
Related Products: Anti-CD45.2-SAP (Cat. #IT-91), Anti-CD117-SAP (Cat. #IT-83)
See Also:
- Czechowicz A et al. Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation. Nat Commun 10:617, 2019.
- Palchaudhuri R et al. Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin. Nat Biotechnol 34:738-745, 2016.