mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblasts
D. Furtado, C. Cortez-Jugo, Y. H. Hung, A. I. Bush, F. Caruso
This paper demonstrates that engineered mRNA can restore NPC1 protein function and correct the pathogenic phenotype in NP-C1 patient fibroblasts.
bioRxiv · Feb 21, 2022Bioengineering