Liu Xiaoling, Sabnis Himalee, Bunting Kevin D, Qu Cheng-Kui
Division of Hematology and Oncology, Department of Medicine, Center for Stem Cell and Regenerative Medicine, Case Comprehensive Cancer Center, Case Western Reserve University, Cleveland, OH 44106, USA.
Adv Hematol. 2012;2012:308252. doi: 10.1155/2012/308252. Epub 2011 Nov 13.
Significant advances in our understanding of the genetic defects and the pathogenesis of juvenile myelomonocytic leukemia (JMML) have been achieved in the last several years. The information gathered tremendously helps us in designing molecular targeted therapies for this otherwise fatal disease. Various approaches are being investigated to target defective pathways/molecules in this disease. However, effective therapy is still lacking. Development of specific target-based drugs for JMML remains a big challenge and represents a promising direction in this field.
在过去几年中，我们对青少年粒单核细胞白血病（JMML）的基因缺陷和发病机制的理解取得了重大进展。所收集的信息极大地帮助我们设计针对这种致命疾病的分子靶向疗法。目前正在研究各种方法来靶向该疾病中存在缺陷的信号通路/分子。然而，仍然缺乏有效的治疗方法。开发针对JMML的特异性靶向药物仍然是一个巨大的挑战，也是该领域一个有前景的方向。
