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Association of genetic variants of ABCA1 with susceptibility to dementia: (SADEM study).
Metab Brain Dis. 2020 Aug;35(6):915-922. doi: 10.1007/s11011-020-00577-4. Epub 2020 May 23.
Metab Brain Dis. 2020.
PMID: 32447570
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience.
Lomash RM, Shchelochkov O, Chandler RJ, Venditti CP, Pariser AR, Ottinger EA; NIH PaVe-GT Team.
Lomash RM, et al.
Hum Gene Ther. 2023 Mar;34(5-6):217-227. doi: 10.1089/hum.2022.232.
Hum Gene Ther. 2023.
PMID: 36694456
Free PMC article.
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