Below are the most recent publications written about "Gene Silencing" by people in Profiles.
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Fakih HH, Ohara M, Summers A, Sarli SL, Kelly K, Gagnon R, Maru B, Bramato B, Khvorova A, Watts JK. Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system. Nucleic Acids Res. 2026 Sep 22; 54(18).
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Belgrad J, Summers A, Landles C, Greene JR, Hildebrand S, Knox E, Sapp E, Yamada N, Furgal R, Miller R, Osborne GF, Chase K, Luu E, Freedman J, Bramato B, McHugh N, Nnah I, Benoit V, O'Reilly D, Greer P, Bates GP, Vogt TF, Lee R, Howland D, DiFiglia M, Aronin N, Khvorova A. Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington's disease pathogenesis in mice. Sci Transl Med. 2026 Aug 12; 18(862):eaea3104.
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Hariharan VN, Summers A, Clipperton-Allen AE, Caiazzi J, Hildebrand SR, O' Reilly D, Tang Q, Kennedy Z, Echeverria D, McHugh N, Cooper D, Sousa J, Ferguson C, Fakih HH, Bogdanik L, Coenraads M, Khvorova A. Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality in an MECP2 duplication mouse model. Nat Commun. 2026 Jul 21; 17(1).
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Wan F, He J, Ma H, PiresFerreira D, Kumanan V, Lee JS, Chen X, He R, Su Q, Gallagher TL, Zhu S, Cabrera GT, Zhao L, Shen J, Gruntman A, Brown RH, Xu Z, Gao G, Xie J. Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model. Nat Commun. 2026 06 25; 17(1).
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Larsen EC, Moon JE, King OD, Lawrence JB. Selective chr21 homolog silencing reveals polymorphisms influence the epigenetic silencing and functional dosage of RWDD2B. Am J Hum Genet. 2026 Apr 02; 113(4):715-735.
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Ho S, Rice NP, Yu T, Weng Z, Theurkauf WE. Aub, Vasa, and Armi concentration in phase-separated nuage is dispensable for piRNA biogenesis and transposon silencing. RNA. 2026 02 17; 32(3):341-358.
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Fakih HH, Zain Ui Abideen M, Rachid MO, Gross KY, Ormsby TJR, Ross VM, Gagnon R, Dahlke C, Furgal RC, Lochmann C, Summers A, Sleiman HF, Fürst SM, Jackson SN, Gordilho JO, Bouix-Peter C, Portal T, Tang Q, Harris JE, Khvorova A, Blanchard C, Alterman JF. Intradermal delivery of lipophilic siRNAs enables prolonged skin retention and sustained gene silencing in a porcine model. Nat Commun. 2026 Feb 03; 17(1).
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O'Reilly D, Furgal RC, Ross VM, Luu E, Gross KY, Hariharan V, Summers A, Cooper D, Allen S, Dahlke C, Rachid MO, Chan IL, Fakih HH, Alterman JF, Echeverria D, Watts JK, Khvorova A. Presence of phosphodiester backbone, but not nucleobases, in the guide's 3' terminal region is necessary for RISC loading and target cleavage in vitro and in vivo. Nucleic Acids Res. 2026 Jan 05; 54(1).
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Fakih HH, Lochmann C, Gagnon R, Summers A, Caiazzi J, Buchwald JE, Tang Q, Maru B, Hildebrand SR, Abideen MZUI, Furgal RC, Gross KY, Yang YS, Cooper D, Monopoli KR, Echeverria D, Shim JH, Yamada K, Alterman JF, Khvorova A. Potent and durable gene modulation in heart and muscle with chemically defined lipophilic siRNAs. Nucleic Acids Res. 2025 11 26; 53(22).
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Nagaraj H, Jeon T, Cicek YA, Goswami R, Nasim N, Mhaske R, Rotello VM. Efficient Cytosolic Delivery of siRNA Using Lyophilized and Reconstituted Polymer-siRNA Polyplexes. Pharm Res. 2025 Dec; 42(12):2369-2377.