Author

E. Bartoszek

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Open access Oct 2026

Generation of a novel knock-in mouse line expressing oncogenic Kit D814V in the hematopoietic stem cell compartment as a preclinical model for systemic mastocytosis.

Research in mastocytosis, characterized by clonal expansion of mast cells (MC), and driven by the KIT D816V mutation in >90% of patients, has been hampered by the lack of in vivo models. Here, we describe a novel knock-in mouse line expressing Kit D814V (murine homolog to human KIT D816V) in the hematopoietic compartme...

S. Stivala, A. Makeeva, Tiago Almeida Fonseca et al. · Leukemia · 0 citations

Open access Jul 2026

CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model

The results suggest that this approach may overcome the reliance on busulfan or other myeloablative conditioning regimens with their associated morbidities, and by enabling toxin-free conditioning and in vivo selection of edited cells, may facilitate clinical implementation of these highly valuable genetic therapies.

Romina Marone, Rosalba Lepore, K. Paschoudi et al. · bioRxiv · 0 citations