Cystinosin deficient rats recapitulate the phenotype of nephropathic cystinosis
J. A. Hollywood, P. K. Kallingappa, P. Y. Cheung, R. M. Martis, S. Sreebhavan, A. Chatterjee, E. J. Buckels, B. G. Matthews, P. M. Lewis, 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.
bioRxiv · Jun 29, 2021Physiology