Every year, the Friedrich-Alexander University Erlangen-Nürnberg carries out a nationwide selection for the recipient of its prestigious Cord-Michael Becker Prize. Amounting to 5000 Euros, the prize was established in 2013 in honour of Professor Cord-Michael Becker, who founded the Molecular Medicine study programme in Erlangen, and is meant to recognize outstanding PhD work in this field. This year, the prize went to Maurice for his thesis: “Functional analysis of the leukemic evolution in children with Down syndrome utilizing CRISPR-Cas9 genome editing”.
As indicated in the title of his thesis, Maurice’s PhD work focused on Down syndrome myeloid leukemia, a rare childhood blood cancer which provides a unique opportunity to study leukemic evolution given its roots in transient abnormal myeloipoiesis (TAM). Nearly all newborns with TAM harbor a genetic mutation in the GATA1 gene. In most cases, patients go into spontaneous remission, but sometimes, TAM develops into full-blown myeloid leukemia after the acquisition of additional mutations. Maurice leveraged the CRISPR-Cas9 genome editing system to model this two-step process, by inducing subsequent mutations in GATA1 and other genes identified from exome sequencing of TAM and ML-DS patients. His research has led to two publications in Nucleic Acids Research and Cancer Cell, on the topics of CRISPR-Cas9 sgRNA efficacy prediction and the evolution of TAM to ML-DS, respectively.