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 (Q24643018)

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English	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	scientific article

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scholarly article

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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 (English)

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Duchenne muscular dystrophy

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Francesco Muntoni

25

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Sebahattin Çırak

4

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Maria Elena Garralda

11

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Virginia Arechavala-Gomeza

2

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Dominic J Wells

object named as

Dominic J Wells

18

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21

object named as

Volker Straub

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20

object named as

Ryszard Kole

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Matthew J. Wood

17

object named as

Matthew J A Wood

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ORCID Public Data File 2021

Petros Nihoyannopoulos

10

object named as

Petros Nihoyannopoulos

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Caroline A Sewry

23

object named as

Caroline Sewry

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author name string

Maria Kinali

1

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Lucy Feng

3

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David Hunt

5

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Carl Adkin

6

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Michela Guglieri

7

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Emma Ashton

8

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Stephen Abbs

9

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Mary Rutherford

12

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Caroline McCulley

13

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Linda Popplewell

14

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Ian R Graham

15

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George Dickson

16

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Steve D Wilton

19

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Kate Bushby

22

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Jennifer E Morgan

24

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language of work or name

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publication date

October 2009

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Lancet Neurology

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8

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10

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918-28

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Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications

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7 April 2017

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

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7 April 2017

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

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27 September 2017

149th ENMC International Workshop and 1st TREAT-NMD Workshop on: "planning phase i/ii clinical trials using systemically delivered antisense oligonucleotides in duchenne muscular dystrophy".

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27 September 2017

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

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27 September 2017

Muscle MRI in inherited neuromuscular disorders: past, present, and future

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27 September 2017

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

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27 September 2017

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

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27 September 2017

Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

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27 September 2017

Application of in vitro Myo-differentiation of non-muscle cells to enhance gene expression and facilitate analysis of muscle proteins

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27 September 2017

Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy

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27 September 2017

The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion

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27 September 2017

Somatic reversion/suppression in Duchenne muscular dystrophy (DMD): evidence supporting a frame-restoring mechanism in rare dystrophin-positive fibers

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27 September 2017

Systemically delivered antisense oligomers upregulate gene expression in mouse tissues

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30 May 2018

Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy

1 reference

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30 May 2018

The use of the social stress and support interview in families with deviant children: methodological issues.

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30 May 2018

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

1 reference

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28 November 2018

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

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28 November 2018

Dystrophin levels as low as 30% are sufficient to avoid muscular dystrophy in the human

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28 November 2018

Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.

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28 November 2018

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2755039

28 November 2018

Long-term expression of full-length human dystrophin in transgenic mdx mice expressing internally deleted human dystrophins

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28 November 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2755039

28 November 2018

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

1 reference

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28 November 2018

A short protocol for muscle MRI in children with muscular dystrophies.

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28 November 2018

Using the Strengths and Difficulties Questionnaire (SDQ) to screen for child psychiatric disorders in a community sample.

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28 November 2018

Elevation of serum creatine kinase as the only manifestation of an intragenic deletion of the dystrophin gene in three unrelated families

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28 November 2018

Validation of the Hospital Anxiety and Depression Scale for use with adolescents

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12 December 2020

based on heuristic

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Muscle MRI findings in a three-generation family affected by Bethlem myopathy

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Computed tomographic patterns of muscles in neuromuscular diseases

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The clinical, genetic and dystrophin characteristics of Becker muscular dystrophy. II. Correlation of phenotype with genetic and protein abnormalities

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A quantitative ELISA for dystrophin

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

[Family study allows more optimistic prognosis and genetic counselling in a child with a deletion of exons 50-51 of the dystrophin gene]

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

https://pubmed.ncbi.nlm.nih.gov/19713152

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Muscle MRI findings in a three-generation family affected by Bethlem myopathy

1 reference

https://api.crossref.org/works/10.1016%2FS1474-4422%2809%2970211-X

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1016/S1474-4422(09)70211-X

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Consolidated OpenCitations Corpus – April 2017

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OpenCitations bibliographic resource ID

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Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

PMC publication ID

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

PubMed publication ID

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

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