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

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17 December 2017

limb-girdle muscular dystrophy

main subject

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author name string

Carinne Roudaut

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17 December 2017

Samia Martin

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17 December 2017

Françoise Fougerousse

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17 December 2017

Laurence Suel

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17 December 2017

Jérôme Poupiot

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17 December 2017

Evelyne Gicquel

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17 December 2017

Fanny Noulet

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17 December 2017

Olivier Danos

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17 December 2017

Isabelle Richard

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17 December 2017

language of work or name

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14 November 2005

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17 December 2017

published in

Molecular Therapy

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17 December 2017

volume

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issue

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page(s)

250-259

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17 December 2017

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

cites work

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

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

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

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

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

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

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

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Identifiers

DOI

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17 December 2017

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17 December 2017