(Q38184276)

30 August 2017

title

Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases (English)

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30 August 2017

author

Annemieke Aartsma-Rus

series ordinal

2

object named as

Annemieke Aartsma-Rus

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author name string

Marcel Veltrop

series ordinal

1

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30 August 2017

language of work or name

English

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publication date

31 January 2014

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Experimental Cell Research

30 August 2017

published in

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30 August 2017

volume

325

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30 August 2017

issue

1

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30 August 2017

page(s)

50-55

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New insights in gene-derived therapy: the example of Duchenne muscular dystrophy

30 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Lack of myostatin results in excessive muscle growth but impaired force generation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Development of multiexon skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

In-frame deletion in a novel centrosomal/ciliary protein CEP290/NPHP6 perturbs its interaction with RPGR and results in early-onset retinal degeneration in the rd16 mouse

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

7 January 2021

Spinal muscular atrophy

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Splicing therapy for neuromuscular disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

AON-mediated Exon Skipping Restores Ciliation in Fibroblasts Harboring the Common Leber Congenital Amaurosis CEP290 Mutation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Lack of myostatin impairs mechanical performance and ATP cost of contraction in exercising mouse gastrocnemius muscle in vivo.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Systemic administration of PRO051 in Duchenne's muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Lack of exon 10 in the murine tau gene results in mild sensorimotor defects with aging

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Targeting RNA splicing for disease therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Fibrodysplasia ossificans progressiva: mechanisms and models of skeletal metamorphosis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Eteplirsen for the treatment of Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Spinal muscular atrophy: manifestations and management

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Genetic abnormalities in fibrodysplasia ossificans progressiva

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

The domestic cat as a large animal model for characterization of disease and therapeutic intervention in hereditary retinal blindness

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Tau alternative splicing in familial and sporadic tauopathies.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Targeting a pre-mRNA structure with bipartite antisense molecules modulates tau alternative splicing

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Antisense oligonucleotides for the treatment of spinal muscular atrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressiva

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Peptide nucleic acid (PNA) cell penetrating peptide (CPP) conjugates as carriers for cellular delivery of antisense oligomers

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Splice modulating therapies for human disease

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Neurodegenerative disorder FTDP-17-related tau intron 10 +16C → T mutation increases tau exon 10 splicing and causes tauopathy in transgenic mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Local dystrophin restoration with antisense oligonucleotide PRO051

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Structural analyses of the pre-mRNA splicing machinery

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

A novel morpholino oligomer targeting ISS-N1 improves rescue of severe spinal muscular atrophy transgenic mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

Regulation of alternative splicing by local histone modifications: potential roles for RNA-guided mechanisms

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YEXCR.2014.01.026

10.1016/J.YEXCR.2014.01.026

7 January 2021

Identifiers

DOI

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30 August 2017

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