A novel Zebrafish model of Kaposiform Lymphangiomatosis: A tool for drug discovery
Awardee: Karina Yaniv
Institution: Sheba Medical Center
Grant Amount: $68,650.00
Funding Period: February 1, 2023 - January 31, 2024
Summary:
Kaposiform lymphangiomatosis (KLA) is a member of a broad family of complex lymphatic anomalies (CLA)- rare disorders characterized by the abnormal proliferation of lymphatic vessels in the skin and internal organs. KLA, the most aggressive and rarest form of these disorders, can occur at any age, but the incidence is highest in children and teenagers. Current pharmaceutical treatments are aimed chiefly at managing the symptoms; thus, the 5-year survival rate for children affected by KLA is only about 50%. Therefore, there is an urgent need for new pre-clinical models recapitulating the disease and enabling the identification of novel drug targets. This study aims to characterize a novel KLA zebrafish model we recently established in our lab and to screen for new avenues of treatment. Because of their small size, transparency, and large progeny, ZF have become an attractive means for assessing compound effects at early stages of drug discovery. Recently, a lifesaving treatment for a lymphatic anomaly was identified through a chemical screen based on our early establishment of the ZF as a superb model for the study of lymphatic biology. Here we will harness the power of our novel mutants to screen for compounds that selectively revert the KLA-related phenotypes. We anticipate that completion of this study will help increase our understanding of the etiology of KLA and will lead to the identification of new efficient therapies.