Margaux Mombled

GENETHOFF is a Snakemake workflow for analyzing CRISPR/Cas9 off-targets from GUIDE-Seq data, handling multiple organisms and Cas nucleases through a metadata-driven approach.

This study uses multiplex base editing to target BCL11A enhancers, increasing fetal hemoglobin production and reducing sickle cell disease symptoms without causing significant genomic damage.

This paper describes a prime editing strategy to modify γ-globin promoters in HSPCs to increase HbF expression for sickle cell disease treatment.

Preclinical safety and efficacy studies of a new gene therapy approach for patients with severe β-hemoglobinopathies.