Inherited predispositions and hyperactive Ras in myeloid leukemogenesis

Pediatr Blood Cancer. 2006 May 1;46(5):579-85. doi: 10.1002/pbc.20644.

Abstract

Identifying the molecular basis for inherited cancer predispositions reveals genes that when mutated, play a critical role in the earliest stages of tumorigenesis. Although rare, inherited predispositions to myeloid leukemias have led to a greater understanding of pathways important for myeloid proliferation and maturation. In particular, elucidating why children with neurofibromatosis type 1 (NF1) and Noonan syndrome (NS) are predisposed to juvenile myelomonocytic leukemia (JMML) has uncovered a critical role of hyperactive Ras signaling in normal myeloid growth and leukemogenesis. Here, we review studies of human samples and experiments performed in genetically engineered strains of mice investigating the molecular and biochemical basis of aberrant growth in JMML. These strains model human disease features and provide an opportunity to investigate novel therapeutic strategies that may ultimately cure JMML and other myeloid malignancies characterized by hyperactive Ras.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Animals
  • Forecasting
  • Genetic Predisposition to Disease*
  • Humans
  • Leukemia, Myeloid / genetics*
  • Leukemia, Myeloid / physiopathology
  • Leukemia, Myelomonocytic, Chronic / genetics
  • Leukemia, Myelomonocytic, Chronic / physiopathology
  • Myeloproliferative Disorders / genetics*
  • Myeloproliferative Disorders / physiopathology
  • Neurofibromatosis 1 / genetics
  • Neurofibromatosis 1 / physiopathology
  • Neurofibromin 1 / physiology
  • Proto-Oncogene Proteins p21(ras) / metabolism*
  • Signal Transduction

Substances

  • Neurofibromin 1
  • Proto-Oncogene Proteins p21(ras)