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Page 1
Sortilin inhibition treats multiple neurodegenerative lysosomal storage disorders.
bioRxiv [Preprint]. 2023 Sep 22:2023.09.22.559064. doi: 10.1101/2023.09.22.559064.
bioRxiv. 2023.
PMID: 37790379
Free PMC article.
Preprint.
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease.
Johnson TB, White KA, Brudvig JJ, Cain JT, Langin L, Pratt MA, Booth CD, Timm DJ, Davis SS, Meyerink B, Likhite S, Meyer K, Weimer JM.
Johnson TB, et al. Among authors: booth cd.
Mol Ther. 2021 Jan 6;29(1):162-175. doi: 10.1016/j.ymthe.2020.09.033. Epub 2020 Sep 24.
Mol Ther. 2021.
PMID: 33010819
Free PMC article.
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Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.
Johnson TB, Brudvig JJ, Likhite S, Pratt MA, White KA, Cain JT, Booth CD, Timm DJ, Davis SS, Meyerink B, Pineda R, Dennys-Rivers C, Kaspar BK, Meyer K, Weimer JM.
Johnson TB, et al. Among authors: booth cd.
Front Genet. 2023 Mar 24;14:1118649. doi: 10.3389/fgene.2023.1118649. eCollection 2023.
Front Genet. 2023.
PMID: 37035740
Free PMC article.
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