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Dysregulation of PAX5 causes uncommitted B cell development and tumorigenesis in mice

Boast, B.; Helian, K.; Andrews, D.; Li, X.; Cho, V.; Closa Mosquera, A.; Sutton, H. J.; Reed, J. H.; Bergmann, H.; Roots, C. M.; Yabas, M.; Barthel, N.; Omari, S. A.; Young, C.; Miosge, L. A.; Eyras, E.; Nutt, S. L.; Hein, N.; Hannan, K. M.; Cockburn, I. A.; Goodnow, C. C.; Enders, A.

2021-01-31 immunology
10.1101/2021.01.29.428877 bioRxiv
Show abstract

PAX5 is the master transcription factor controlling B cell identity. In humans, mutations in PAX5 account for 30% of B cell acute lymphoblastic leukemia (B-ALL) cases. Investigating the causal effects of PAX5 mutations has however been difficult due to the premature lethality of Pax5-/- mice. Here we describe a novel mouse strain with a premature STOP mutation in Pax5 (Y351*) that produces a truncated protein and reduction in protein function, yet still allows for some B cell development to occur. A population of uncommitted and multipotent CD19+B220- B cells develops in the bone marrow of homozygous mice leading to the development of B-ALL. We show that the tumors frequently acquire secondary mutations in Jak3, and Ptpn11 highlighting key pathways interacting with PAX5 during malignant transformation. Analysis of the PAX5Y351* mice provide insight not only into the functional consequence of reduced PAX5 activity on B cell development and identity, but also provides an avenue in which to study PAX5-driven B-ALL in mice. One Sentence SummaryReduction in PAX5 function in mice induces the development of uncommitted B cells that have multipotent and malignant potential.

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