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Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

JAMA neurology, 2020-08, Vol.77 (8), p.982

2020

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Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides

The New England journal of medicine, 2017-07, Vol.377 (3), p.222-232

2017

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RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges

Pharmacological reviews, 2020-10, Vol.72 (4), p.862-898

2020

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Pharmacology of Antisense Drugs

Annual review of pharmacology and toxicology, 2017-01, Vol.57 (1), p.81-105

2017

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RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

Annual review of pharmacology and toxicology, 2010-02, Vol.50 (1), p.259-293

2010

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Safety of antisense oligonucleotide and siRNA-based therapeutics

Drug discovery today, 2017-05, Vol.22 (5), p.823-833

2017

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Therapeutic Antisense Oligonucleotides Are Coming of Age

Annual review of medicine, 2019-01, Vol.70 (1), p.307-321

Bennett, C Frank

2019

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Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study

The Lancet (British edition), 2015-10, Vol.386 (10002), p.1472-1483

2015

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Chemical modification and design of anti-miRNA oligonucleotides

Gene therapy, 2011-12, Vol.18 (12), p.1111-1120

2011

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Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics

Molecular therapy, 2021-02, Vol.29 (2), p.540-554

2021

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Nkx6.1 and nkx6.2 regulate α- and β-cell formation in zebrafish by acting on pancreatic endocrine progenitor cells

Developmental biology, 2010-04, Vol.340 (2), p.397-407

2010

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Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

Neuron (Cambridge, Mass.), 2016-05, Vol.90 (3), p.535-550

2016

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A framework for individualized splice-switching oligonucleotide therapy

Nature (London), 2023-07, Vol.619 (7971), p.828-836

2023

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Opportunities and challenges for antisense oligonucleotide therapies

Journal of inherited metabolic disease, 2021-01, Vol.44 (1), p.72-87

2021

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Towards a therapy for Angelman syndrome by targeting a long non-coding RNA

Nature (London), 2015-02, Vol.518 (7539), p.409-412

2015

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Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

Proceedings of the National Academy of Sciences - PNAS, 2013-11, Vol.110 (47), p.E4530-E4539

2013

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Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

Nucleic acids research, 2022-06, Vol.50 (10), p.5443-5466

2022

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Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

Nature communications, 2020-07, Vol.11 (1), p.3501-13, Article 3501

2020

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Differential uptake, kinetics and mechanisms of intracellular trafficking of next-generation antisense oligonucleotides across human cancer cell lines

Nucleic acids research, 2019-05, Vol.47 (9), p.4375-4392

2019

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Cryo-EM and antisense targeting of the 28-kDa frameshift stimulation element from the SARS-CoV-2 RNA genome

Nature structural & molecular biology, 2021-09, Vol.28 (9), p.747-754

2021

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Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans

Circulation research, 2013-05, Vol.112 (11), p.1479-1490

2013

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Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases

Neuron (Cambridge, Mass.), 2017-06, Vol.94 (6), p.1056-1070

2017

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Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Nature medicine, 2022-01, Vol.28 (1), p.104-116

2022

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Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency

Proceedings of the National Academy of Sciences - PNAS, 2020-12, Vol.117 (51), p.32370-32379

2020

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A transfer-RNA-derived small RNA regulates ribosome biogenesis

Nature (London), 2017-12, Vol.552 (7683), p.57-62

2017

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Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides

RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges

Pharmacology of Antisense Drugs

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

Safety of antisense oligonucleotide and siRNA-based therapeutics

Therapeutic Antisense Oligonucleotides Are Coming of Age

Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study

Chemical modification and design of anti-miRNA oligonucleotides

Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics

Nkx6.1 and nkx6.2 regulate α- and β-cell formation in zebrafish by acting on pancreatic endocrine progenitor cells

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

A framework for individualized splice-switching oligonucleotide therapy

Opportunities and challenges for antisense oligonucleotide therapies

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression

Differential uptake, kinetics and mechanisms of intracellular trafficking of next-generation antisense oligonucleotides across human cancer cell lines

Cryo-EM and antisense targeting of the 28-kDa frameshift stimulation element from the SARS-CoV-2 RNA genome

Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans

Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases

Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis

Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency

A transfer-RNA-derived small RNA regulates ribosome biogenesis

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