Guillaume Corre

This paper introduces TILV, a CRISPR-assisted method for precise targeted integration of transgenes in human hematopoietic stem cells using IDLV donors, showing improved efficiency and minimal impact on cell viability.

This paper describes a novel rat model with an exon 45 deletion in the Dmd gene, showing features of DMD and exon skipping, which may inform personalized therapies.

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.

The paper develops a liver-targeted gene therapy using recombinant AAV8 to cure Lysosomal acid lipase deficiency by correcting lipid accumulation and normalizing gene expression and mitochondrial function in a mouse model.

This study identifies dysregulated miRNAs in DMD patients and mice, linking cholesterol metabolism and the mevalonate pathway to disease progression, with Simvastatin treatment showing therapeutic potential.