(Q43268180)

30 June 2018

title

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

1 reference

30 June 2018

limb-girdle muscular dystrophy

main subject

gene therapy

0 references

1 reference

autosomal recessive limb-girdle muscular dystrophy type 2D

inferred from title

1 reference

5

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30 June 2018

author name string

Jerry R Mendell

1

1 reference

30 June 2018

Louise R Rodino-Klapac

2

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30 June 2018

Xiomara Rosales-Quintero

3

1 reference

30 June 2018

Janaiah Kota

4

1 reference

30 June 2018

Gloria Galloway

6

1 reference

30 June 2018

Josepha M Craenen

7

1 reference

30 June 2018

Sarah Lewis

8

1 reference

30 June 2018

Vinod Malik

9

1 reference

30 June 2018

Christopher Shilling

10

1 reference

30 June 2018

Barry J Byrne

11

1 reference

30 June 2018

Thomas Conlon

12

1 reference

30 June 2018

Katherine J Campbell

13

1 reference

30 June 2018

William G Bremer

14

1 reference

30 June 2018

Laurence Viollet

15

1 reference

30 June 2018

Christopher M Walker

16

1 reference

30 June 2018

Zarife Sahenk

17

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30 June 2018

K Reed Clark

18

1 reference

30 June 2018

language of work or name

1 reference

1 September 2009

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30 June 2018

published in

Annals of Neurology

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30 June 2018

volume

66

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30 June 2018

issue

3

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30 June 2018

page(s)

290-297

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30 June 2018

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Sarcoglycan subcomplex in normal and pathological human muscle fibers

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

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

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Molecular and cell biology of the sarcoglycan complex

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Novel adeno-associated virus vector vaccine restricts replication of simian immunodeficiency virus in macaques

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Limb-girdle muscular dystrophies--from genetics to molecular pathology

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Homozygous alpha-sarcoglycan mutation in two siblings: one asymptomatic and one steroid-responsive mild limb-girdle muscular dystrophy patient

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Mutational diversity and hot spots in the alpha-sarcoglycan gene in autosomal recessive muscular dystrophy (LGMD2D)

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

E-box sites and a proximal regulatory region of the muscle creatine kinase gene differentially regulate expression in diverse skeletal muscles and cardiac muscle of transgenic mice

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

A common missense mutation in the adhalin gene in three unrelated Brazilian families with a relatively mild form of autosomal recessive limb-girdle muscular dystrophy

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Sarcoglycanopathies: can muscle immunoanalysis predict the genotype?

3 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Delivery of alpha- and beta-sarcoglycan by recombinant adeno-associated virus: efficient rescue of muscle, but differential toxicity

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors

15 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=6014624

Correlation of muscle biopsy, clinical course, and outcome in PM and sporadic IBM

15 August 2018

1 reference

12 December 2020

Limb-girdle muscular dystrophy in the United States

1 reference

12 December 2020

Primary alpha-sarcoglycan deficiency responsive to immunosuppression over three years

1 reference

12 December 2020

Phenotypic Correction of α-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector.

1 reference

12 December 2020

Identifiers

DOI

10.1002/ANA.21732

1 reference

30 June 2018

PMCID

6014624

1 reference

30 June 2018

PubMed ID

19798725

1 reference

30 June 2018