卡波西样淋巴管瘤病候选治疗药物

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斑马鱼模型助力寻找罕见淋巴病潜在疗法
Xin Hua She· 2025-09-12 07:09
Core Insights - Researchers at the Weizmann Institute of Science have discovered a potential new treatment pathway for Kaposi's sarcoma-like lymphangioendothelioma, a rare genetic disease, using zebrafish embryos due to their biological similarities to humans [1][2] - The disease causes abnormal expansion and deformation of lymphatic vessels, leading to severe respiratory difficulties and multi-organ involvement, with no effective treatments currently available [1] - The research team introduced NRAS gene mutations found in patient cells into zebrafish embryos, successfully creating a disease model that closely resembles human conditions [1] - The study revealed that the mutated NRAS gene remains "stuck" in an active state, causing unlimited proliferation of lymphatic cells [1] - An automated drug screening system was developed, testing approximately 150 approved drugs, resulting in the identification of two candidate drugs that significantly reversed cardiac and lymphatic abnormalities in zebrafish models [1] - These candidate drugs demonstrated higher safety and lower side effects compared to some existing anti-cancer drugs targeting NRAS [1] Future Directions - Due to the rarity of the disease, the research team plans to collaborate across centers to expedite clinical trials, aiming to provide new treatment options for patients [2]