Author
National Institutes of HealthORCID 0000-0001-6322-8991Unclaimed ORCID profile · follow to get notified of new preprints
4 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.
M. Kyle Cromer, Joab Camarena, Renata M. Martin…·Matthew Hebden Porteus
This study replaces the HBA1 gene with HBB in β-thalassemia-derived HSPCs using CRISPR, normalizing globin ratios and restoring functional hemoglobin.
Juan Pablo Ruíz, Guibin Chen, Juan J. Haro‐Mora…·André Larochelle
This study develops a scalable monolayer culture system for generating erythroid and multilineage hematopoietic progenitors from human iPSCs, showing limited HSC engraftment potential.
Diego A. Espinoza, Xing Fan, Di Yang…·Cynthia E. Dunbar
This paper reports a case of clonal hematopoiesis in a rhesus macaque following lentiviral transduction of HSPC with a strong retroviral promoter, leading to gene overexpression and aberrant splicing.