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Precision Medicine in Myelodysplastic Syndromes and Leukemias: Lessons from Sequential Mutations
Annual Review of Medicine ( IF 10.5 ) Pub Date : 2017-01-18 00:00:00 , DOI: 10.1146/annurev-med-062915-095637
Aziz Nazha 1 , Mikkael A. Sekeres 1
Affiliation  

Precision medicine can be simply defined as the identification of personalized treatment that matches patient-specific clinical and genomic characteristics. Since the completion of the Human Genome Project in 2003, significant advances have been made in our understanding of the genetic makeup of diseases, especially cancers. The identification of somatic mutations that can drive cancer has led to the development of therapies that specifically target the abnormal proteins derived from these mutations. This has led to a paradigm shift in our treatment methodology. Although some success has been achieved in targeting some genetic abnormalities, several challenges and limitations exist when applying precision-medicine concepts in leukemia and myelodysplastic syndromes. We review the current understanding of genomics in myelodysplastic syndromes (MDS) and leukemias and the limitations of precision-medicine concepts in MDS.

中文翻译:


骨髓增生异常综合症和白血病中的精准医学:顺序突变的经验教训

精确医学可以简单地定义为与患者特定的临床和基因组特征相匹配的个性化治疗方法。自2003年人类基因组计划完成以来,我们对疾病(尤其是癌症)的基因组成的理解取得了重大进展。可以驱动癌症的体细胞突变的鉴定导致了针对特定于源自这些突变的异常蛋白的疗法的发展。这导致我们的治疗方法发生了范式转变。尽管在针对某些遗传异常方面已经取得了一些成功,但是在白血病和骨髓增生异常综合症中应用精确医学概念时,仍然存在一些挑战和局限性。

更新日期:2017-01-18
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