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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
1969 1
1976 2
1978 1
1979 1
1980 2
1981 1
1982 5
1983 1
1984 4
1985 3
1986 2
1987 3
1988 3
1989 3
1990 9
1991 2
1992 5
1993 4
1995 5
1996 2
1997 5
1998 3
1999 5
2000 3
2001 6
2002 5
2003 3
2004 2
2005 5
2006 1
2007 2
2008 2
2009 6
2010 6
2011 2
2012 11
2013 6
2014 6
2015 5
2016 8
2017 4
2018 9
2019 6
2020 4
2021 4
2022 4
2023 10
2024 8

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192 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.
Huntington disease.
Vonsattel JP, DiFiglia M. Vonsattel JP, et al. Among authors: difiglia m. J Neuropathol Exp Neurol. 1998 May;57(5):369-84. doi: 10.1097/00005072-199805000-00001. J Neuropathol Exp Neurol. 1998. PMID: 9596408 Review. No abstract available.
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Conroy F, Miller R, Alterman JF, Hassler MR, Echeverria D, Godinho BMDC, Knox EG, Sapp E, Sousa J, Yamada K, Mahmood F, Boudi A, Kegel-Gleason K, DiFiglia M, Aronin N, Khvorova A, Pfister EL. Conroy F, et al. Among authors: difiglia m. Nat Commun. 2022 Oct 3;13(1):5802. doi: 10.1038/s41467-022-33061-x. Nat Commun. 2022. PMID: 36192390 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 Nov 1;31(11):3355-3356. doi: 10.1016/j.ymthe.2023.09.016. Epub 2023 Sep 25. Mol Ther. 2023. PMID: 37751745 Free article. No abstract available.
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
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
Hariharan VN, Caiazzi J, Miller R, Ferguson C, Sapp E, Fakih H, Tang Q, Yamada N, Furgal R, Paquette J, Bramato B, McHugh N, Summers A, Lochmann C, Godinho B, Hildebrand S, Echeverria D, Hassler M, Alterman J, DiFiglia M, Aronin N, Khvorova A, Yamada K. Hariharan VN, et al. Among authors: difiglia m. Res Sq [Preprint]. 2023 Jun 1:rs.3.rs-2987323. doi: 10.21203/rs.3.rs-2987323/v1. Res Sq. 2023. Update in: Nat Biotechnol. 2024 Aug 1. doi: 10.1038/s41587-024-02336-7. PMID: 37398145 Free PMC article. Updated. Preprint.
An early start to Huntington's disease.
DiFiglia M. DiFiglia M. Science. 2020 Aug 14;369(6505):771-772. doi: 10.1126/science.abd6215. Science. 2020. PMID: 32792383 No abstract available.
192 results