M. Kyle Cromer

UNCOVERseq is a workflow for sensitive off-target nomination in CRISPR-Cas editing, demonstrating superior performance across multiple modalities and systems.

This paper develops a combinatorial base editing strategy to enhance erythroid lineage amplification and disease correction in hematopoietic stem and progenitor cells.

This study demonstrates efficient in utero and neonatal lipid nanoparticle delivery for genome editing of hematopoietic stem cells, achieving robust editing and multilineage reconstitution.

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

This paper describes a genome editing strategy that restores hemoglobin production in α-thalassemia major-derived hematopoietic stem and progenitor cells by integrating a functional α-globin gene and a truncated erythropoietin receptor.

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.

The paper uses ultra-deep sequencing to assess oncogenic mutations in human hematopoietic stem and progenitor cells after CRISPR/Cas9 editing, finding no evidence of tumorigenic variants.

This study replaces the HBA1 gene with HBB in β-thalassemia-derived HSPCs using CRISPR, normalizing globin ratios and restoring functional hemoglobin.