Article
Human gene-engineered calreticulin mutant stem cells recapitulate MPN hallmarks and identify targetable vulnerabilities.
Leukemia - 1 Apr 2023
Foßelteder Johannes, Pabst Gabriel, Sconocchia Tommaso, Schlacher Angelika, Auinger Lisa, Kashofer Karl, Beham-Schmid Christine, Trajanoski Slave, Waskow Claudia, Schöll Wolfgang, Sill Heinz, Zebisch Armin, Wölfler Albert, Thomas Daniel, Reinisch Andreas
Abstract excerpt
Calreticulin (CALR) mutations present the main oncogenic drivers in JAK2 wildtype (WT) myeloproliferative neoplasms (MPN), including essential thrombocythemia and myelofibrosis, where mutant (MUT) CALR is increasingly recognized as a suitable mutation-specific drug target. However, our current understanding of its mechanism-of-action is derived from mouse models or immortalized cell lines, where cross-species...
Topics
- Humans
- Animals
- Mice
- Calreticulin
- Janus Kinase 2
- Myeloproliferative Disorders
- Mutation
- Hematopoietic Stem Cells
- Primary Myelofibrosis