Below are the most recent publications written about "Oligonucleotides, Antisense" by people in Profiles.
-
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.
-
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.
-
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.
-
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.
-
Meijboom KE, Brown RH. Approaches to Gene Modulation Therapy for ALS. Neurotherapeutics. 2022 07; 19(4):1159-1179.
-
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.
-
Khvorova A. Modulation of DNA transcription: The future of ASO therapeutics? Cell. 2022 06 09; 185(12):2011-2013.
-
Tran H, Moazami MP, Yang H, McKenna-Yasek D, Douthwright CL, Pinto C, Metterville J, Shin M, Sanil N, Dooley C, Puri A, Weiss A, Wightman N, Gray-Edwards H, Marosfoi M, King RM, Kenderdine T, Fabris D, Bowser R, Watts JK, Brown RH. Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide. Nat Med. 2022 01; 28(1):117-124.
-
Shin M, Meda Krishnamurthy P, Devi G, Watts JK. Quantification of Antisense Oligonucleotides by Splint Ligation and Quantitative Polymerase Chain Reaction. Nucleic Acid Ther. 2022 02; 32(1):66-73.
-
Lidberg KA, Annalora AJ, Jozic M, Elson DJ, Wang L, Bammler TK, Ramm S, Monteiro MB, Himmelfarb J, Marcus CB, Iversen PL, Kelly EJ. Antisense oligonucleotide development for the selective modulation of CYP3A5 in renal disease. Sci Rep. 2021 02 25; 11(1):4722.