(Q24643018)

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

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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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inferred from title

Duchenne muscular dystrophy

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inferred from title

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

1 reference

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

1 reference

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7 April 2017

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

1 reference

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

7 April 2017

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

1 reference

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

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".

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

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

1 reference

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

27 September 2017

Systemically delivered antisense oligomers upregulate gene expression in mouse tissues

1 reference

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

30 May 2018

Impact of nasal ventilation on survival in hypercapnic Duchenne muscular dystrophy

1 reference

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

30 May 2018

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

1 reference

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

30 May 2018

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

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

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

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

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

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

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

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

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

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

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

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

28 November 2018

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

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

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

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

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

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

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

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://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

1 reference

Consolidated OpenCitations Corpus – April 2017

OpenCitations bibliographic resource ID

OpenCitations bibliographic resource ID

1 reference

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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