Tatjana I. Cornu

CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates β-thalassemia model

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

Next-generation all-in-one CRISPR/Cas9 multiply-edited CD30CAR-T cells:Potency despite risk of translocations

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

Castling, a novel therapeutic concept for rewiring pathological gene-expression networks, enabled by the TRIPLE technology

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