Associated Papers
Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies.
Azaria RD et al.
Jci Insight, 2024
Alterations in Proteostasis Mechanisms in Niemann-Pick Type C Disease.
Servín Muñoz IV et al.
International Journal of Molecular Sciences, 2024
Species-specific differences in NPC1 protein trafficking govern therapeutic response in Niemann-Pick type C disease.
Schultz ML et al.
Jci Insight, 2022
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