Roman M. Doll

This study uses CRISPR-Cas genome editing to selectively inactivate JAK2 V617F mutations in patient cells, showing that heterozygous cells are not oncogene addicted while homozygous cells are eradicated, suggesting genome editing as a potential treatment for MPNs.

This study identifies Rab35 as a tumor suppressor that modulates Wnt signaling through a GTPase relay, highlighting its role in restraining intestinal tumorigenesis.

This paper describes the optimization of base editing to correct the SMN2 exon 7 mutation in SMA, achieving high editing efficiency and increased SMN protein levels in vitro and in vivo.

This paper develops a temperature-tolerant CRISPR base editor for efficient and precise gene inactivation in Drosophila, showing high efficiency and minimal byproducts.