骨髓增生异常综合征的分子畸变

Advances in cell and gene therapy Pub Date : 2020-04-13 DOI:10.1002/acg2.83

Nikesh N. Shah, Rami S. Komrokji

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引用次数: 0

摘要

32曲美替尼是一种经常用于黑色素瘤的RAS和MEK抑制剂，也已在NRAS和KRAS突变的高危MDS中进行了研究。33 APR-246（Aprea Therapeutics）是一种与突变的TP53共价结合并恢复其正常功能的药物，正在与阿扎胞苷联合测试高危MDS和具有TP53突变的AML患者，28 Ib/II期研究显示出有希望的结果，完全缓解率为60%-80%，III期研究目前正在进行中。34

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Molecular aberrations in myelodysplastic syndromes