Kun Jia

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 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 uses a bacteriophage-derived peptide to control CBE activity, improving targeting precision and gene correction efficiency in a Marfan syndrome model.