Panagiotis Antoniou

The paper analyzes CRISPR-Cas9 off-target mutations and translocations at single-cell and tissue-specific levels in mice, revealing context-dependent variability in editing outcomes.

This paper demonstrates that replacing the BCL11A repressor binding site with a TAL1:GATA1 activator motif in HBG1/2 promoters enhances γ-globin reactivation and corrects sickle cell disease in hematopoietic stem/progenitor cells.

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.

This study evaluates the safety and efficacy of CRISPR/Cas9-mediated HbF reactivation in sickle cell disease by testing it in patient-derived HSPCs and shows it is effective but has off-target effects.

This paper improves prime editing by modulating DNA repair pathways and engineering pegRNA, achieving higher precision and efficiency in human and mouse cells.