Understanding and Treating Niemann-Pick Type C Disease: Models Matter.
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SUBMITTER: Pallottini V
PROVIDER: S-EPMC7730076 | biostudies-literature | 2020 Nov
REPOSITORIES: biostudies-literature
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Understanding and Treating Niemann-Pick Type C Disease: Models Matter.
Pallottini Valentina V Pfrieger Frank W FW
International journal of molecular sciences 20201126 23
Biomedical research aims to understand the molecular mechanisms causing human diseases and to develop curative therapies. So far, these goals have been achieved for a small fraction of diseases, limiting factors being the availability, validity, and use of experimental models. Niemann-Pick type C (NPC) is a prime example for a disease that lacks a curative therapy despite substantial breakthroughs. This rare, fatal, and autosomal-recessive disorder is caused by defects in NPC1 or NPC2. These ubi ...[more]
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