Article
Potential clinical use of azacitidine and MEK inhibitor combination therapy in PTPN11-mutated juvenile myelomonocytic leukemia.
Molecular therapy : the journal of the American Society of Gene Therapy - 5 Apr 2023
Pasupuleti Santhosh Kumar, Chao Karen, Ramdas Baskar, Kanumuri Rahul, Palam Lakshmi Reddy, Liu Sheng, Wan Jun, Annesley Colleen, Loh Mignon L, Stieglitz Elliot, Burke Michael J, Kapur Reuben
Abstract excerpt
Juvenile myelomonocytic leukemia (JMML) is a rare myeloproliferative neoplasm of childhood. The molecular hallmark of JMML is hyperactivation of the Ras/MAPK pathway with the most common cause being mutations in the gene PTPN11, encoding the protein tyrosine phosphatase SHP2. Current strategies for treating JMML include using the hypomethylating agent, 5-azacitidine (5-Aza) or MEK inhibitors trametinib and...
Topics
- Animals
- Mice
- Azacitidine
- Leukemia, Myelomonocytic, Juvenile
- Mitogen-Activated Protein Kinase Kinases
- Mutation
- Protein Kinase Inhibitors
- Protein Tyrosine Phosphatase, Non-Receptor Type 11
- Humans