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Advances in Hematology
Volume 2012, Article ID 308252, 7 pages
Review Article

Molecular Targets for the Treatment of Juvenile Myelomonocytic Leukemia

1Division 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
2Aflac Cancer Center of Children's Health Care of Atlanta and, Department of Pediatrics, Emory University, Atlanta, GA 30322, USA

Received 13 May 2011; Revised 13 July 2011; Accepted 11 August 2011

Academic Editor: Michael H. Tomasson

Copyright © 2012 Xiaoling Liu et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.


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.