(Q45888604)

17 December 2017

title

Safety and efficacy of AAV-mediated calpain 3 gene transfer in a mouse model of limb-girdle muscular dystrophy type 2A. (English)

1 reference

17 December 2017

limb-girdle muscular dystrophy

main subject

1 reference

1

1 reference

17 December 2017

author name string

Carinne Roudaut

2

1 reference

17 December 2017

Samia Martin

3

1 reference

17 December 2017

Françoise Fougerousse

4

1 reference

17 December 2017

Laurence Suel

5

1 reference

17 December 2017

Jérôme Poupiot

6

1 reference

17 December 2017

Evelyne Gicquel

7

1 reference

17 December 2017

Fanny Noulet

8

1 reference

17 December 2017

Olivier Danos

9

1 reference

17 December 2017

Isabelle Richard

10

1 reference

17 December 2017

language of work or name

1 reference

14 November 2005

1 reference

17 December 2017

published in

Molecular Therapy

1 reference

17 December 2017

volume

13

1 reference

17 December 2017

issue

2

1 reference

17 December 2017

page(s)

250-259

1 reference

17 December 2017

Mutations in the proteolytic enzyme calpain 3 cause limb-girdle muscular dystrophy type 2A

cites work

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Calpainopathy-a survey of mutations and polymorphisms

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Chromosome 15-linked limb-girdle muscular dystrophy: clinical phenotypes in Reunion Island and French metropolitan communities

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Loss of calpain 3 proteolytic activity leads to muscular dystrophy and to apoptosis-associated IkappaBalpha/nuclear factor kappaB pathway perturbation in mice.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Force impairment in calpain 3-deficient mice is not correlated with mechanical disruption.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Muscle-specific calpain, p94, responsible for limb girdle muscular dystrophy type 2A, associates with connectin through IS2, a p94-specific sequence

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Calpain 3 is activated through autolysis within the active site and lyses sarcomeric and sarcolemmal components

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Insertion sequence 1 of muscle-specific calpain, p94, acts as an internal propeptide.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Down-regulation of genes in the lysosomal and ubiquitin-proteasome proteolytic pathways in calpain-3-deficient muscle.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Calpains in muscle wasting.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Optimal design of a single recombinant adeno-associated virus derived from serotypes 1 and 2 to achieve more tightly regulated transgene expression from nonhuman primate muscle

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Efficient expression of naked dna delivered intraarterially to limb muscles of nonhuman primates

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Expression and functional characteristics of calpain 3 isoforms generated through tissue-specific transcriptional and posttranscriptional events

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Characterization of monoclonal antibodies to calpain 3 and protein expression in muscle from patients with limb-girdle muscular dystrophy type 2A

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Null mutation of calpain 3 (p94) in mice causes abnormal sarcomere formation in vivo and in vitro

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Normal calpain expression in genetically confirmed limb-girdle muscular dystrophy type 2A

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

MUSEAP, a novel reporter gene for the study of long-term gene expression in immunocompetent mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Intracellular trafficking of adeno-associated virus vectors: routing to the late endosomal compartment and proteasome degradation

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

Factors influencing recombinant adeno-associated virus production

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2005.09.017

10.1016/J.YMTHE.2005.09.017

7 January 2021

Identifiers

DOI

1 reference

17 December 2017

1 reference

17 December 2017