PhysiologybioRxiv
Cystinosin deficient rats recapitulate the phenotype of nephropathic cystinosis
J. A. Hollywood, P. K. Kallingappa, P. Y. Cheung…·A. J. Davidson
This paper develops a new cystinosis rat model using CRISPR/Cas9 to recapitulate the disease phenotype and progression more accurately than existing models.
DOI 10.1152/ajprenal.00277.2021