US EditionNovel research into AADC enzyme may help advance treatment
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Novel research into AADC enzyme may help advance treatment
Scientists created a new cell model of AADC deficiency that has allowed them to understand exactly how two disease-causing mutations in the DDC gene affect the AADC enzyme. According to the team, the new model “is useful to study the molecular basis of the disease.” It “recapitulates some key features of AADC deficiency, … and represents an ideal system for small molecule screening regarding specific enzyme defects, paving the way for a precisio…
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