Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies.
Azaria RD, Correia AB, Schache KJ, Zapata M, Pathmasiri KC, Mohanty V, Nannapaneni DT, Ashfeld BL, Helquist P, Wiest O, Ohgane K, Li Q, Fredenburg RA, Blagg BS, Cologna SM, Schultz ML, Lieberman AP.
Azaria RD, et al. Among authors: correia ab.
JCI Insight. 2024 Aug 29;9(20):e179525. doi: 10.1172/jci.insight.179525.
JCI Insight. 2024.
PMID: 39207850
Free PMC article.