Panagiotis Antoniou

Single-Cell and Tissue-Specific CRISPR Editing Analyses Unveil New Insights to Off-Targets and Translocations

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.

Enhanced γ-globin reactivation and sickle cell correction through a repressor-to- activator motif switch in the HBG1/2 promoters

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.

Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease

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.

A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease

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

Base editing of β0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies

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

Improved nuclease-based prime editing by DNA repair modulation and pegRNA engineering

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