Figure 5: Xenograft recapitulates the clonal changes occurring in RARS patient bone marrow compartment.

Mutational analysis of sequential primary TNC (total nucleated cells) sample from one patient (MDS1). Primary patient sample was received at two different time points, MDS1 bone marrow cells (TP1, first sample) and MDS1 bone marrow cells (TP2, second sample). Mutational analysis was also performed on human engrafted cells, which were obtained from mice transplanted with the patient sample received at time point 1. The length of the arc represents the MABs. Gene MAB for human engrafted cells is the average between three animal experiments. Three independent PCR/sequencing experiments were performed to confirm/determine the mutant allele burden throughout the experiments. Error Bar in the bar chart were derived from the PCR/sequencing technical replicates. MAB, mutant allele burden; TP, time point; HEC, human engrafted cells; BM, bone marrow cells.