mRNA Degradation as a Therapeutic Solution for Mucopolysaccharidosis Type IIIC: Use of Antisense Oligonucleotides to Promote Downregulation of Heparan Sulfate Synthesis. [PDF]
Santos JI+11 more
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Antisense Oligonucleotides for Rapid Translation of Gene Therapy in Glioblastoma. [PDF]
Desgraves JF+9 more
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Sequence-Specific Free Energy Changes in DNA/RNA Induced by a Single LNA-T Modification in Antisense Oligonucleotides. [PDF]
Tomita-Sudo E+4 more
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mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs). [PDF]
Susorov D+3 more
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Antisense oligonucleotides provide optimism to the therapeutic landscape for tauopathies. [PDF]
Harris GA, Hirschfeld LR.
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Therapeutic potential of APP antisense oligonucleotides for Alzheimer's disease and down syndrome-related Alzheimer's disease. [PDF]
Thirumalai S, Patani R, Hung C.
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Characterizing the plasma protein binding profiles of chemistry diversified antisense oligonucleotides in human and mouse plasma using an ultrafiltration method. [PDF]
Yun C, Fukami K, Shinkyo R, Jiang RR.
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<i>In vivo</i> efficacy and safety of systemically administered serinol nucleic acid-modified antisense oligonucleotides in mouse kidney. [PDF]
Tsuboi T+12 more
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Exploring non-coding variants and evaluation of antisense oligonucleotides for splicing redirection in Usher syndrome. [PDF]
García-Bohórquez B+14 more
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Phase 1/2 trial of brogidirsen: Dual-targeting antisense oligonucleotides for exon 44 skipping in Duchenne muscular dystrophy. [PDF]
Komaki H+10 more
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