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Year Number of Results
2001 1
2002 4
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2004 2
2005 5
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2007 1
2008 2
2009 6
2010 6
2011 2
2012 11
2013 6
2014 6
2015 5
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112 results

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Page 1
Allele-selective lowering of mutant HTT protein by HTT-LC3 linker compounds.
Li Z, Wang C, Wang Z, Zhu C, Li J, Sha T, Ma L, Gao C, Yang Y, Sun Y, Wang J, Sun X, Lu C, Difiglia M, Mei Y, Ding C, Luo S, Dang Y, Ding Y, Fei Y, Lu B. Li Z, et al. Among authors: difiglia m. Nature. 2019 Nov;575(7781):203-209. doi: 10.1038/s41586-019-1722-1. Epub 2019 Oct 30. Nature. 2019. PMID: 31666698
A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Alterman JF, et al. Among authors: difiglia m. Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2. Nat Biotechnol. 2019. PMID: 31375812 Free PMC article.
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease.
O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, Monopoli K, Yang XW, Carroll J, DiFiglia M, Aronin N, Khvorova A. O'Reilly D, et al. Among authors: difiglia m. Mol Ther. 2023 Jun 7;31(6):1661-1674. doi: 10.1016/j.ymthe.2023.05.006. Epub 2023 May 12. Mol Ther. 2023. PMID: 37177784 Free PMC article.
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones.
Yamada K, Hariharan VN, Caiazzi J, Miller R, Ferguson CM, Sapp E, Fakih HH, Tang Q, Yamada N, Furgal RC, Paquette JD, Biscans A, Bramato BM, McHugh N, Summers A, Lochmann C, Godinho BMDC, Hildebrand S, Jackson SO, Echeverria D, Hassler MR, Alterman JF, DiFiglia M, Aronin N, Khvorova A. Yamada K, et al. Among authors: difiglia m. Nat Biotechnol. 2024 Aug 1. doi: 10.1038/s41587-024-02336-7. Online ahead of print. Nat Biotechnol. 2024. PMID: 39090305
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system.
Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, Khvorova A. Belgrad J, et al. Among authors: difiglia m. Nucleic Acids Res. 2024 Jun 24;52(11):6099-6113. doi: 10.1093/nar/gkae368. Nucleic Acids Res. 2024. PMID: 38726879 Free PMC article.
Towards Standardizing Nomenclature in Huntington's Disease Research.
DiFiglia M, Leavitt BR, Macdonald D, Thompson LM; Huntington’s Disease Nomenclature Working Group:. DiFiglia M, et al. J Huntingtons Dis. 2024;13(2):119-131. doi: 10.3233/JHD-240044. J Huntingtons Dis. 2024. PMID: 38968054 Free PMC article. Review.
Nacc1 Mutation in Mice Models Rare Neurodevelopmental Disorder with Underlying Synaptic Dysfunction.
Deehan MA, Kothuis JM, Sapp E, Chase K, Ke Y, Seeley C, Iuliano M, Kim E, Kennington L, Miller R, Boudi A, Shing K, Li X, Pfister E, Anaclet C, Brodsky M, Kegel-Gleason K, Aronin N, DiFiglia M. Deehan MA, et al. Among authors: difiglia m. J Neurosci. 2024 Apr 3;44(14):e1610232024. doi: 10.1523/JNEUROSCI.1610-23.2024. J Neurosci. 2024. PMID: 38388424 Free PMC article.
112 results