Below are the most recent publications written about "Oligonucleotides, Antisense" by people in Profiles.
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Susorov D, Echeverria D, Khvorova A, Korostelev AA. mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). Nucleic Acids Res. 2024 Aug 27; 52(15):8687-8701.
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Sarli SL, Fakih HH, Kelly K, Devi G, Rembetsy-Brown JM, McEachern HR, Ferguson CM, Echeverria D, Lee J, Sousa J, Sleiman HF, Khvorova A, Watts JK. Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo. Nucleic Acids Res. 2024 May 22; 52(9):4799-4817.
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Engelbeen S, O'Reilly D, Van De Vijver D, Verhaart I, van Putten M, Hariharan V, Hassler M, Khvorova A, Damha MJ, Aartsma-Rus A. Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy. Nucleic Acid Ther. 2023 12; 33(6):348-360.
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Shah S, Sharp KJ, Raju Ponny S, Lee J, Watts JK, Berry-Kravis E, Richter JD. Antisense oligonucleotide rescue of CGG expansion-dependent FMR1 mis-splicing in fragile X syndrome restores FMRP. Proc Natl Acad Sci U S A. 2023 07 04; 120(27):e2302534120.
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Goyenvalle A, Jimenez-Mallebrera C, van Roon W, Sewing S, Krieg AM, Arechavala-Gomeza V, Andersson P. Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides. Nucleic Acid Ther. 2023 01; 33(1):1-16.
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Aartsma-Rus A, Garanto A, van Roon-Mom W, McConnell EM, Suslovitch V, Yan WX, Watts JK, Yu TW. Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic Acid Ther. 2023 01; 33(1):17-25.
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Wang F, Calvo-Roitberg E, Rembetsy-Brown JM, Fang M, Sousa J, Kartje ZJ, Krishnamurthy PM, Lee J, Green MR, Pai AA, Watts JK. G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects. Nucleic Acids Res. 2022 12 09; 50(22):12657-12673.
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Meijboom KE, Brown RH. Approaches to Gene Modulation Therapy for ALS. Neurotherapeutics. 2022 07; 19(4):1159-1179.
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Shin M, Chan IL, Cao Y, Gruntman AM, Lee J, Sousa J, Rodr?guez TC, Echeverria D, Devi G, Debacker AJ, Moazami MP, Krishnamurthy PM, Rembetsy-Brown JM, Kelly K, Yukselen O, Donnard E, Parsons TJ, Khvorova A, Sontheimer EJ, Maehr R, Garber M, Watts JK. Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts. Nucleic Acids Res. 2022 08 26; 50(15):8418-8430.
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Khvorova A. Modulation of DNA transcription: The future of ASO therapeutics? Cell. 2022 06 09; 185(12):2011-2013.