Toni Cathomen

CRISCross improves CRISPR-Cas9 off-target prediction by integrating epigenetic features with sequence data across cell types, showing better performance than sequence-only models.

This paper demonstrates a method using CD117-blocking antibody and epitope-engineered HSPCs to treat β-thalassemia without myeloablative conditioning.

This paper develops gene-edited tacrolimus-resistant Treg cells (FKBP12KO-Treg) for clinical use in transplant recipients, showing they maintain immunoregulatory function while resisting tacrolimus.

This study develops next-generation CD30CAR-T cells using CRISPR/Cas9 for multiplex editing, showing high potency but risks of translocations with combined knockouts.

The paper introduces a therapeutic strategy called castling to correct gene-expression imbalances by rewiring miRNA regulation using the TRIPLE genome-editing technology.

This paper develops BEKI, a base editor-based method for targeted transgene insertion and multiplex gene disruption in T cells, reducing chromosomal translocations compared to Cas9-based approaches.

This paper develops a non-viral CRISPR-Cas12a method to redirect Tregs with CARs by integrating an antigen-binding domain into the CD3 epsilon gene, showing improved antigen-specific function and graft protection in a mouse model.

This paper develops a method to integrate ζ-deficient CARs into the CD3ζ gene to create functional CAR-T and CAR-NK cells with improved safety and efficacy for off-the-shelf therapies.

The paper validates off-target effects of CRISPR/Cas9 in rhesus macaques using error-corrected sequencing, showing that in vitro and in silico methods have limited predictive accuracy.

This paper demonstrates efficient gene editing in FA mouse models using TALENs and a donor template at a safe harbor locus, achieving phenotypic correction of FA cells.