Joab Camarena

The paper introduces SPIT, a method using human cells to deliver genetic tools in vivo, demonstrating its application for modifying multiple organs and tissues in immunocompetent mice.

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 uses gene editing in hematopoietic stem cells to treat Pyruvate Kinase Deficiency Hemolytic Anemia, restoring RPK function and ATP levels in erythroid cells.

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

This study corrects the beta-globin gene in sickle cell disease patient-derived HSCs using CRISPR/Cas9 and shows improved in vivo erythropoiesis in a mouse model.

The paper introduces TRACE-Seq, a method to track clonal reconstitution of gene-targeted human hematopoietic stem cells using barcoded AAV6 donor templates in immunodeficient mice.

This paper introduces a Cas9/AAV6 system for high-frequency genome editing in human pluripotent stem cells without selection markers, achieving up to 94% integration efficiency for large DNA cassettes.