BioengineeringbioRxiv
mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblasts
D. Furtado, C. Cortez-Jugo, Y. H. Hung…·F. Caruso
This paper demonstrates that engineered mRNA can restore NPC1 protein function and correct the pathogenic phenotype in NP-C1 patient fibroblasts.
DOI 10.1021/acs.molpharmaceut.2c00463