BioengineeringbioRxivPreprint
Gene surgery as a potential treatment option for Nephropathic Cystinosis in vitro
E. Sendino Garvi, J. Faria, C. Pou Casellas…·M. J. Janssen
This study uses CRISPR/Cas9 HITI to repair CTNS in ciPTEC cells, achieving functional cystinosin restoration and reversing the cystinotic phenotype.
DOI 10.1101/2023.11.01.565117