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The FDA's pharmacy panel currently has a number of vacancies, which Kennedy could fill before the July meeting
Silence or correct mutations: e.g., SNCA and LRRK2 via RNA interference (RNAi) or CRISPR-Cas9 ( AAV2-based vectors have been used in clinical trials to deliver therapeutic genes to the striatum or SNpc, showing safety and moderate benefit ( 5.5 Stem cell-based approaches Stem cell therapies offer the potential to replace lost dopaminergic neurons and restore neural circuitry
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doi: 10.4049/jimmunol.168.4.1618 51 Johnson-LgerCAAurrand-LionsMBeltraminelliNFaselNImhofBA
doi: 10.1084/jem.20170355 21 ThomasEDRambergRESaleGESparkesRSGoldeDW