Dystrophin immunity in Duchenne's muscular dystrophy. (Q41590387)

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15 February 2020

title

Dystrophin immunity in Duchenne's muscular dystrophy (English)

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12

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author name string

Jerry R Mendell

1

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15 February 2020

Katherine Campbell

2

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15 February 2020

Louise Rodino-Klapac

3

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Zarife Sahenk

4

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15 February 2020

Chris Shilling

5

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15 February 2020

Sarah Lewis

6

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Dawn Bowles

7

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Steven Gray

8

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Chengwen Li

9

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Gloria Galloway

10

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15 February 2020

Vinod Malik

11

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15 February 2020

K Reed Clark

13

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Juan Li

14

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Xiao Xiao

15

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Jade Samulski

16

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15 February 2020

Scott W McPhee

17

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R Jude Samulski

18

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Christopher M Walker

19

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15 February 2020

language of work or name

English

0 references

publication date

1 October 2010

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The New England Journal of Medicine

15 February 2020

published in

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volume

363

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issue

15

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15 February 2020

page(s)

1429-1437

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

15 February 2020

cites work

1 reference

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

6 June 2018

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AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

6 June 2018

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T cell-mediated autoimmune disease due to low-affinity crossreactivity to common microbial peptides

6 June 2018

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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

6 June 2018

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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

6 June 2018

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Effect of gene therapy on visual function in Leber's congenital amaurosis

6 June 2018

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Safety and efficacy of gene transfer for Leber's congenital amaurosis

6 June 2018

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Genetic treatments in muscular dystrophies

6 June 2018

1 reference

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The role of corticosteroids in muscular dystrophy: a critical appraisal

6 June 2018

1 reference

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rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

6 June 2018

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

6 June 2018

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Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy

6 June 2018

1 reference

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Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

6 June 2018

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Analysis of T cell receptor repertoire of muscle-infiltrating T lymphocytes in polymyositis. Restricted V alpha/beta rearrangements may indicate antigen-driven selection

6 June 2018

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Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy

6 June 2018

1 reference

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

6 June 2018

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

6 June 2018

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CD8(+) T-cell responses to adeno-associated virus capsid in humans

19 August 2018

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Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice

19 August 2018

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Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy

19 August 2018

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Local dystrophin restoration with antisense oligonucleotide PRO051

19 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/20925545

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Specific T cell receptor gene rearrangements at the site of muscle degeneration in Duchenne muscular dystrophy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/20925545

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The muscular dystrophies

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/20925545

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1056/NEJMOA1000228

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:20925545%20AND%20SRC:MED&resulttype=core&format=json

15 February 2020

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15 February 2020

PubMed publication ID

20925545

1 reference