Kiran R. Majeti

This paper develops small molecule-inducible EPO receptors to enable erythropoiesis without recombinant EPO, reducing costs and improving scalability of RBC manufacturing.

The paper uses human genetics and CRISPR/Cas9 to recreate a naturally occurring mutation that increases red blood cell production and shows its combination with a beta-thalassemia correction strategy enhances RBC development.

This study compares CRISPR off-target detection tools using ex vivo edited CD34+ HSPCs, finding that HiFi Cas9 reduces off-target sites and some methods outperform others in sensitivity and specificity.