Results 41 to 50 of about 89,699 (157)
Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis
Annals of Clinical and Translational NeurologyAntisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been ...
Daisuke Ito, Kensuke Okada
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F1000Research, 2019 Recent approvals of oligonucleotide analogue drugs to alter gene expression have been welcomed by patient communities but not universally supported. These compounds represent a class of drugs that are designed to target a specific gene transcript, and ...
Ianthe Pitout +3 more
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Pharmacokinetics of antisense oligonucleotide drugs
Фармакокинетика и Фармакодинамика, 2013 This review covers the pharmacokinetic characteristics of the various antisense oligonucleotide drugs. A comparison of the pharmacokinetics of drugs first and second generation.
M. R. Khaitov, V. V. Smirnov
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Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease
Molecules, 2017 Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting.
Valentina Sardone +4 more
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Journal of Plant Protection Research, 2019 5.8S ribosomal RNA plays an important role in protein synthesis and eukaryotic ribosome translocation. Contact DNA insecticides based on antisense fragments of 5.8S ribosomal RNA gene of gypsy moth Lymantria dispar L.
Volodymyr V. Oberemok +9 more
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Molecular Therapy: Nucleic Acids, 2019 Eluforsen (previously known as QR-010) is a 33-mer 2′-O-methyl modified phosphorothioate antisense oligonucleotide targeting the F508del mutation in the gene encoding CFTR protein of cystic fibrosis patients.
Jaeah Kim +6 more
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ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy
Translational Neuroscience, 2017 Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene.
Ottesen Eric W.
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Molecular Therapy: Nucleic Acids, 2017 Antisense oligonucleotide (AON) therapeutics offer new avenues to pursue clinically relevant targets inaccessible with other technologies. Advances in improving AON affinity and stability by incorporation of high affinity nucleotides, such as locked ...
Annie Moisan +17 more
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Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders
University of Ottawa Journal of Medicine, 2018 Antisense oligonucleotides (ASOs) are synthetic, single-stranded DNA molecules that can bind to specific mRNA sequences and alter protein expression.
Ryan Gotesman
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Meeting report: antisense oligonucleotides.
Haematologica, 1994 The use of antisense oligonucleotides as a therapeutic tool in modulating gene expression represents a newly established strategy for treating diseases. Such oligomers may be designed to complement a region of a specific gene or messenger RNA. Using this approach, oligonucleotides can serve as a potential block of transcription or translation through ...
G, Martinelli, S, Ferrari
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