Assessment of FDA-Approved Drugs as a Therapeutic Approach for Niemann-Pick Disease Type C1 Using Patient-Specific iPSC-Based Model Systems

Assessment of FDA-Approved Drugs as a Therapeutic Approach for Niemann-Pick Disease Type C1 Using Patient-Specific iPSC-Based Model Systems

Niemann-Pick type C1 (NP-C1) is a fatal, progressive neurodegenerative disease caused by mutations in the <i>NPC1</i> gene. Mutations of <i>NPC1</i> can result in a misfolded protein that is subsequently marked for proteasomal degradation. Such loss-of-function mutations lead...

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Bibliographic Details
Main Authors: Christin Völkner, Supansa Pantoom, Maik Liedtke, Jan Lukas, Andreas Hermann, Moritz J. Frech
Format: Article
Language:English
Published: MDPI AG 2022-01-01
Series:Cells
Subjects:
pharmacological chaperones
NPC1
in silico screening
molecular docking
induced pluripotent stem cells
Online Access:https://www.mdpi.com/2073-4409/11/3/319

Internet

https://www.mdpi.com/2073-4409/11/3/319

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