Gene surgery as a potential treatment option for Nephropathic Cystinosis in vitro
E. Sendino Garvi, J. Faria, C. Pou Casellas, S. Thijssen, E. J. Wubbolts, A. Jamalpoor, P. Harrison, R. Masereeuw, M. J. Janssen
This study uses CRISPR/Cas9 HITI to repair CTNS in ciPTEC cells, achieving functional cystinosin restoration and reversing the cystinotic phenotype.
bioRxiv · Nov 1, 2023 · PreprintBioengineering