(Q34633598)

3 August 2017

title

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector (English)

1 reference

Duchenne muscular dystrophy

3 August 2017

0 references

1 reference

inferred from title

author

Mavis Agbandje-McKenna

13

object named as

Mavis Agbandje-McKenna

1 reference

3 August 2017

Dawn E Bowles

1

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3 August 2017

Scott W J McPhee

2

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3 August 2017

Chengwen Li

3

1 reference

3 August 2017

Steven J Gray

4

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3 August 2017

Jade J Samulski

5

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3 August 2017

Angelique S Camp

6

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3 August 2017

Juan Li

7

1 reference

3 August 2017

Bing Wang

8

1 reference

3 August 2017

Paul E Monahan

9

1 reference

3 August 2017

Joseph E Rabinowitz

10

1 reference

3 August 2017

Joshua C Grieger

11

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3 August 2017

Lakshmanan Govindasamy

12

1 reference

3 August 2017

Xiao Xiao

14

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3 August 2017

R Jude Samulski

15

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3 August 2017

publication date

8 November 2011

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3 August 2017

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3 August 2017

volume

20

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3 August 2017

issue

2

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3 August 2017

page(s)

443-455

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Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results

3 August 2017

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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7 July 2018

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Dystrophin immunity in Duchenne's muscular dystrophy.

7 July 2018

1 reference

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Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3277234

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26 October 2018

1 reference

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A canine minidystrophin is functional and therapeutic in mdx mice

26 October 2018

1 reference

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26 October 2018

1 reference

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26 October 2018

1 reference

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Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3277234

Gene transfer into skeletal muscle using novel AAV serotypes.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3277234

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3277234

26 October 2018

The muscular dystrophies

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22068425

12 December 2020

inferred from PubMed ID database lookup

Systemic human minidystrophin gene transfer improves functions and life span of dystrophin and dystrophin/utrophin-deficient mice

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22068425

12 December 2020

inferred from PubMed ID database lookup

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FMT.2011.237

7 January 2021

inferred from DOI database lookup

Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle.

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FMT.2011.237

7 January 2021

inferred from DOI database lookup

Identifiers

DOI

10.1038/MT.2011.237

1 reference

3 August 2017

1 reference

3 August 2017

1 reference