(Q47438944)

16 January 2018

title

Making sense of antisense oligonucleotides: A narrative review (English)

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16 January 2018

author name string

Neelam Goyal

series ordinal

1

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16 January 2018

Pushpa Narayanaswami

series ordinal

2

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16 January 2018

publication date

3 November 2017

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16 January 2018

published in

Muscle and Nerve

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16 January 2018

Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide

cites work

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Antisense oligonucleotides in therapy for neurodegenerative disorders

21 January 2018

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RNA therapeutics: RNAi and antisense mechanisms and clinical applications

21 January 2018

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Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

21 January 2018

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Stable reduction of thymidine kinase activity in cells expressing high levels of anti-sense RNA.

21 January 2018

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Fomivirsen: clinical pharmacology and potential drug interactions

21 January 2018

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Multiple evidence strands suggest that there may be as few as 19,000 human protein-coding genes

21 January 2018

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What can exome sequencing do for you?

21 January 2018

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Deep intronic mutations and human disease

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Coupling mRNA processing with transcription in time and space.

21 January 2018

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Initial sequencing and analysis of the human genome

21 January 2018

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Mechanisms of alternative pre-messenger RNA splicing

21 January 2018

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Therapeutic potential of splice-switching oligonucleotides

21 January 2018

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Pre-mRNA splicing and human disease

21 January 2018

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Overview of alternative oligonucleotide chemistries for exon skipping

21 January 2018

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A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new ground

21 January 2018

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Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases

21 January 2018

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Interferon Induction Increased through Chemical Modification of a Synthetic Polyribonucleotide

21 January 2018

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Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide

21 January 2018

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Antisense technologies. Improvement through novel chemical modifications

21 January 2018

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Morpholino antisense oligomers: design, preparation, and properties

21 January 2018

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RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

21 January 2018

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Ribonuclease H: the enzymes in eukaryotes

21 January 2018

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Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs

21 January 2018

1 reference

RNA cleavage products generated by antisense oligonucleotides and siRNAs are processed by the RNA surveillance machinery

21 January 2018

1 reference

Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain

21 January 2018

1 reference

Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron

21 January 2018

1 reference

Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: removal of the CAG containing exon

21 January 2018

1 reference

Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides

21 January 2018

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The medical genetics of dystrophinopathies: molecular genetic diagnosis and its impact on clinical practice

21 January 2018

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RNA-targeted splice-correction therapy for neuromuscular disease

21 January 2018

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Dystrophin and mutations: one gene, several proteins, multiple phenotypes

21 January 2018

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Identification and characterization of a spinal muscular atrophy-determining gene

21 January 2018

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Regulation of SMN protein stability

21 January 2018

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Copy Number Variations in the Survival Motor Neuron Genes: Implications for Spinal Muscular Atrophy and Other Neurodegenerative Diseases

21 January 2018

1 reference

Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy

21 January 2018

1 reference

The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.

21 January 2018

1 reference

The delivery of therapeutic oligonucleotides

21 January 2018

1 reference

Biopharmaceutical drug targeting to the brain

21 January 2018

1 reference

Cell-penetrating peptides: mechanisms and applications

21 January 2018

1 reference

Harnessing the capacity of cell-penetrating peptides for drug delivery to the central nervous system

21 January 2018

1 reference

Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum.

21 January 2018

1 reference

Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals

21 January 2018

1 reference

Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

21 January 2018

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Detection of 98% of DMD/BMD gene deletions by polymerase chain reaction

21 January 2018

1 reference

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

21 January 2018

1 reference

The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations

21 January 2018

1 reference

Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence

21 January 2018

1 reference

Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides

21 January 2018

1 reference

Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells

21 January 2018

1 reference

Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle

21 January 2018

1 reference

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

21 January 2018

1 reference

Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides

21 January 2018

1 reference

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

1 reference

Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

1 reference

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

21 January 2018

1 reference

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

21 January 2018

1 reference

Exon skipping: a first in class strategy for Duchenne muscular dystrophy

21 January 2018

1 reference

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

21 January 2018

1 reference

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

21 January 2018

1 reference

Eteplirsen for the treatment of Duchenne muscular dystrophy

21 January 2018

1 reference

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

21 January 2018

1 reference

Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy.

21 January 2018

1 reference

Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy

21 January 2018

1 reference

Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice

21 January 2018

1 reference

Antisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophy

21 January 2018

1 reference

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

21 January 2018

1 reference

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

21 January 2018

1 reference

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice

21 January 2018

1 reference

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

21 January 2018

1 reference

Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy

21 January 2018

1 reference

A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

21 January 2018

1 reference

Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy

21 January 2018

1 reference

Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.

21 January 2018

1 reference

Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response

21 January 2018

1 reference

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

21 January 2018

1 reference

Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy

21 January 2018

1 reference

An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients

21 January 2018

1 reference

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

21 January 2018

1 reference

The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability

21 January 2018

1 reference

Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS

21 January 2018

1 reference

A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD

21 January 2018

1 reference

Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

21 January 2018

1 reference

State of play in amyotrophic lateral sclerosis genetics

21 January 2018

1 reference

Current hypotheses for the underlying biology of amyotrophic lateral sclerosis

21 January 2018

1 reference

Antisense oligonucleotide therapy for neurodegenerative disease.

21 January 2018

1 reference

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

21 January 2018

1 reference

RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention

21 January 2018

1 reference

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

21 January 2018

1 reference

Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion

21 January 2018

1 reference

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

21 January 2018

1 reference

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

21 January 2018

1 reference

TDP-43 is a developmentally regulated protein essential for early embryonic development

21 January 2018

1 reference

Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3' end of a transcript encoding a protein kinase family member.

21 January 2018

1 reference

Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2.

21 January 2018

1 reference

Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy

21 January 2018

1 reference

Increased steady-state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylation

21 January 2018

1 reference

Loss of the muscle-specific chloride channel in type 1 myotonic dystrophy due to misregulated alternative splicing

21 January 2018

1 reference

Expanded CUG repeats trigger aberrant splicing of ClC-1 chloride channel pre-mRNA and hyperexcitability of skeletal muscle in myotonic dystrophy

21 January 2018

1 reference

Viral vector producing antisense RNA restores myotonic dystrophy myoblast functions

21 January 2018

1 reference

Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy.

21 January 2018

1 reference

Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

21 January 2018

1 reference

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

21 January 2018

1 reference

Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides

21 January 2018

1 reference

Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment.

21 January 2018

1 reference

Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis

21 January 2018

1 reference

Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2

21 January 2018

1 reference

A unifying genetic model for facioscapulohumeral muscular dystrophy

21 January 2018

1 reference

Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics

21 January 2018

1 reference

Antisense targeting of 3' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach.

21 January 2018

1 reference

A novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides

21 January 2018

1 reference

Activating frataxin expression by repeat-targeted nucleic acids

21 January 2018

1 reference

A novel feed-forward loop between ARIH2 E3-ligase and PABPN1 regulates aging-associated muscle degeneration

21 January 2018

1 reference

Ameliorating pathogenesis by removing an exon containing a missense mutation: a potential exon-skipping therapy for laminopathies.

21 January 2018

1 reference

Antisense oligonucleotide-mediated exon skipping of CHRNA1 pre-mRNA as potential therapy for Congenital Myasthenic Syndromes

21 January 2018

1 reference

Antisense-induced messenger depletion corrects a COL6A2 dominant mutation in Ullrich myopathy

21 January 2018

1 reference

Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

21 January 2018

1 reference

Translational and regulatory challenges for exon skipping therapies

21 January 2018

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21 January 2018

Identifiers

DOI

10.1002/MUS.26001

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16 January 2018

PubMed ID

29105153

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16 January 2018