A therapeutic approach to pantothenate kinase associated neurodegeneration

Volume: 9, Issue: 1
Published: Oct 23, 2018
Abstract
Pantothenate kinase (PANK) is a metabolic enzyme that regulates cellular coenzyme A (CoA) levels. There are three human PANK genes, and inactivating mutations in PANK2 lead to pantothenate kinase associated neurodegeneration (PKAN). Here we performed a library screen followed by chemical optimization to produce PZ-2891, an allosteric PANK activator that crosses the blood brain barrier. PZ-2891 occupies the pantothenate pocket and engages the...
Paper Details
Title
A therapeutic approach to pantothenate kinase associated neurodegeneration
Published Date
Oct 23, 2018
Volume
9
Issue
1
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