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This work provides the early tools to develop substrate reduction inhibitors for a genetic childhood seizure disorder, with the hypothesis to target the enzyme (AASS) upstream of the defective gene (ALDH7A1) in the human lysine metabolic pathway. This TEP package includes recombinant human AASS purification protocols, structures of the AASS second domain in different states, in vitro assays to detect ligand binding (differential scanning fluorimetry) and enzyme activity (NADH formation) of human AASS, as well as initial chemical matters.
This document represents version 6 of the TEP datasheet and includes all updates on the project as of April 2019. For more information about TEPs and the TEP Programme, please visit https://thesgc.org/tep.
disease, structure discovery, infectious disease, malaria, chemical probe, chemical biology, structural genomics, metabolic diseases, drug target, drug discovery, neurological genetic disorders, Drug Discovery, Chemical Biology, oncology, target enabling package, cancer, Structural Genomics, neuropsychiatry, neuro, AASS, structure, orphan disease, protein
disease, structure discovery, infectious disease, malaria, chemical probe, chemical biology, structural genomics, metabolic diseases, drug target, drug discovery, neurological genetic disorders, Drug Discovery, Chemical Biology, oncology, target enabling package, cancer, Structural Genomics, neuropsychiatry, neuro, AASS, structure, orphan disease, protein
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