The Norwegian Cancer Society has granted our lab NOK 7 970 000 for developing CRISPR-Cas gene therapy for blood cancer predisposition syndromes for 2022-2025.
The study proposes to develop CRISPR-based therapies for two rare childhood diseases (ADA2 deficiency and Nijmegen Breakage Syndrome) that make the patients susceptible to developing cancer.
In many of these types of cancer predisposition syndromes, the gene editing process can be problematic due to gene defects affecting DNA repair.
Our study investigates this with the aim of developing alternative strategies to overcome risks of unexpected gene editing outcomes, with the hope that the results could also then be applied to other DNA repair diseases.
We are extremely happy that the Norwegian Cancer Society chose to fund our research into these rare childhood diseases. The research enabled by the grant will build further on the excellent preliminary data produced by our group members so far, and will allow us to hire more people into the group for another 3-4 years.
– Pavel Kopcil, Monika Szymanska, Ganna Reint and Jacob Conradi have all put in an enormous effort for setting up the mouse stem cell xenotransplant protocols that are necessary for conducting the proposed studies.