TRACKING HEMATOPOIETIC STEM CELL EVOLUTION IN A WISKOTT-ALDRICH CLINICAL TRIAL
Danilo Pellin, Luca Biasco, Serena Scala…·Ernst C. Wit
This paper develops a statistical model to infer hematopoietic stem cell dynamics in a gene therapy trial for Wiskott-Aldrich Syndrome and finds evidence for a myeloid-based developmental pathway.