Rasmus Otkjær Bak

This study identifies cholesterol biosynthesis as a therapeutic target in CEBPA-mutant AML, showing that statins enhance chemosensitivity in cells with co-mutations in TET2 or WT1.

This paper edits the ADA2 p.R169Q mutation in human hematopoietic stem cells using CRISPR-Cas9 and HDR, showing improved efficiency with NHEJ inhibitors but increased chromosomal deletions.

This study improves CRISPR-Cas12a knock-in efficiency in human T cells by using single-stranded DNA templates with double-stranded Cas-target-sequences, achieving up to 90% knock-in rates for transgene inserts.

The paper uses lineage tracing and scRNA-seq to show that human iPSC differentiation predominantly generates left ventricular cardiomyocytes from the first heart field.

This paper improves prime editing by modifying the reverse transcriptase component to enhance efficiency and enable delivery via split AAV vectors.

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

This study uses CRISPR/Cas9 to edit hematopoietic stem cells to express iduronidase, correcting Mucopolysaccharidosis type I in mice.