Author
St. Jude Children's Research HospitalORCID 0000-0003-3618-2363Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
Recent·Most cited
Varun Katta, Kiera O’Keefe, Yichao Li…·Shengdar Q. Tsai
This study develops a genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease, showing efficient editing, therapeutic HbF levels, and no off-target effects.
Mitchell L. Leibowitz, Stamatis Papathanasiou, Phillip A. Doerfler…·David S. Pellman
This paper shows that CRISPR-Cas9-induced DNA breaks can lead to chromothripsis, a catastrophic chromosomal rearrangement, which is an on-target toxicity in genome editing.
Özge Karayel, Peng Xu, Isabell Bludau…·Matthias Mann
This study uses proteomics to analyze dynamic phospho-signaling networks in human erythropoiesis, identifying key regulatory mechanisms and markers across differentiation stages.