Progression and perspectives in disease modeling for Juvenile myelomonocytic leukemia.

Abstract

Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative neoplasm occurring in infants and young children. JMML has been shown to be resistant to all conventional cytotoxic chemotherapy drugs, and current curative therapies still rely on hematopoietic stem cell transplantation, which carries a high risk of relapse post-transplantation. This underscores the urgent need for novel treatment strategies. However, the rarity of JMML poses a major limitation for research, as it is difficult to collect substantial primary research material. To gain a deeper insight into the underlying biological mechanisms of JMML, researchers are continuously improving and developing preclinical research models to better emulate the disease. Therefore, this review aims to delineate the various experimental models currently employed in JMML, including patient-derived cell-based models, cell models, and animal models. We will discuss the characterization of these models in the context of JMML, hoping to provide a valuable reference for researchers in this field.