The research carried out in the laboratory of Pier Paolo Pandolfi, MD, PhD,has been seminal to elucidating the molecular mechanisms and the genetics underlying the pathogenesis of leukemias, lymphomas and solid tumors as well as in modeling these cancers in the mouse. The Pandolfi laboratory has characterized the function of the fusion oncoproteins and the genes involved in the chromosomal translocations of acute promyelocytic leukemia (APL), as well as of major tumor suppressors such as PTEN and p53, and novel cancer genes such as POKEMON and INPP4B.
The elucidation of the molecular basis underlying APL pathogenesis has led to the development of novel and effective therapeutic strategies. As a result of these efforts, APL is now considered a curable disease. Additional novel therapeutic concepts have emerged from this work and are currently being tested in clinical trials. More recently, Dr. Pandolfi and colleagues have presented a new theory describing how mRNAs, both coding and non-coding, exert their biological functions with profound implications for human genetics, cell biology and cancer biology.