Joab Camarena

Secreted Particle Information Transfer (SPIT) - A Cellular Platform For In Vivo Genetic Engineering

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.

Using human genetics to develop strategies to increase erythropoietic output from genome-edited hematopoietic stem and progenitor cells

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.

Clinically Relevant Gene Editing in Hematopoietic Stem Cells for the Treatment of Pyruvate Kinase Deficiency Hemolytic Anemia

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.

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

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

Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in Mice

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.

TRACE-Seq Reveals Clonal Reconstitution Dynamics of Gene Targeted Human Hematopoietic Stem Cells

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.

Selection-free, high frequency genome editing by homologous recombination of human pluripotent stem cells using Cas9 RNP and AAV6

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.