TY - RPRT TI - mRNA Treatment Rescues Niemann-Pick Disease Type C1 in Patient Fibroblasts AU - Furtado, D. AU - Cortez-Jugo, C. AU - Hung, Y. H. AU - Bush, A. I. AU - Caruso, F. PY - 2022 DO - 10.1101/2022.02.21.479058 UR - https://www.biorxiv.org/content/10.1101/2022.02.21.479058v1 ID - 10.1101/2022.02.21.479058 ER -