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Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Meyer K, et al. Among authors: ferraiuolo l. Mol Ther. 2015 Mar;23(3):477-87. doi: 10.1038/mt.2014.210. Epub 2014 Oct 31. Mol Ther. 2015. PMID: 25358252 Free PMC article.
Micro-RNAs Shuttled by Extracellular Vesicles Secreted from Mesenchymal Stem Cells Dampen Astrocyte Pathological Activation and Support Neuroprotection in In-Vitro Models of ALS.
Provenzano F, Nyberg S, Giunti D, Torazza C, Parodi B, Bonifacino T, Usai C, Kerlero de Rosbo N, Milanese M, Uccelli A, Shaw PJ, Ferraiuolo L, Bonanno G. Provenzano F, et al. Among authors: ferraiuolo l. Cells. 2022 Dec 4;11(23):3923. doi: 10.3390/cells11233923. Cells. 2022. PMID: 36497181 Free PMC article.
Loss of TMEM106B exacerbates C9ALS/FTD DPR pathology by disrupting autophagosome maturation.
Bauer CS, Webster CP, Shaw AC, Kok JR, Castelli LM, Lin YH, Smith EF, Illanes-Álvarez F, Higginbottom A, Shaw PJ, Azzouz M, Ferraiuolo L, Hautbergue GM, Grierson AJ, De Vos KJ. Bauer CS, et al. Among authors: ferraiuolo l. Front Cell Neurosci. 2022 Dec 16;16:1061559. doi: 10.3389/fncel.2022.1061559. eCollection 2022. Front Cell Neurosci. 2022. PMID: 36619668 Free PMC article.
A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export reduces the neurotoxic effects of dipeptide repeat proteins.
Castelli LM, Lin YH, Sanchez-Martinez A, Gül A, Mohd Imran K, Higginbottom A, Upadhyay SK, Márkus NM, Rua Martins R, Cooper-Knock J, Montmasson C, Cohen R, Walton A, Bauer CS, De Vos KJ, Mead RJ, Azzouz M, Dominguez C, Ferraiuolo L, Shaw PJ, Whitworth AJ, Hautbergue GM. Castelli LM, et al. Among authors: ferraiuolo l. Sci Transl Med. 2023 Mar;15(685):eabo3823. doi: 10.1126/scitranslmed.abo3823. Epub 2023 Mar 1. Sci Transl Med. 2023. PMID: 36857431
Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.
Meyer K, Ferraiuolo L, Miranda CJ, Likhite S, McElroy S, Renusch S, Ditsworth D, Lagier-Tourenne C, Smith RA, Ravits J, Burghes AH, Shaw PJ, Cleveland DW, Kolb SJ, Kaspar BK. Meyer K, et al. Among authors: ferraiuolo l. Proc Natl Acad Sci U S A. 2014 Jan 14;111(2):829-32. doi: 10.1073/pnas.1314085111. Epub 2013 Dec 30. Proc Natl Acad Sci U S A. 2014. PMID: 24379375 Free PMC article.
72 results