MOLECULAR THERAPY NUCLEIC ACIDS · Published 2026-07-01 · DOI 10.1016/j.omtn.2026.103022
Ellen R. Koufer, Alice Townsend, Youngseo Na, Adele B. Correia, Noah K. Latham, Koralege C. Pathmasiri, Olga Ilnytska, Judith Storch, Kyle A. Sullivan, Troy Halseth, Thaddeus J. Kunkel, Elizabeth S. Phillippi, Matthew L. Lieberman, Stephanie M. Cologna, Andrew P. Lieberman, Daniel A. Jacobson, Anna S. Schwendeman, Mark L. Schultz
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Koufer, E., Townsend, A., Na, Y., et al. (2026). Codon-Optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo. MOLECULAR THERAPY NUCLEIC ACIDS. https://doi.org/10.1016/j.omtn.2026.103022