Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)
C. Le Guiner
(1, 2)
,
L. Servais
(3)
,
M. Montus
(1)
,
F. Bodvael
(1, 2)
,
B. Gjata
(1)
,
J. Y. Hogrel
(4)
,
P. Carlier
(5, 6)
,
S. Moullec
(7)
,
C. Masurier
(1)
,
O. Adjali
(2)
,
F. Mingozzi
(1)
,
T. Koo
(8)
,
T. Athanasopoulos
(9)
,
Yan Cherel
(10)
,
F. Mavilio
(1)
,
T. Voit
(11, 12)
,
P. Moullier
(13, 14)
,
G. Dickson
(8)
1
Généthon
2 UMR 1089, Atlantic Gene Therapies
3 Service of Clinical Trials and Databases
4 Neuromuscular Physiology and Evaluation Laboratory
5 Institut de Myologie
6 AIM
7 Atlantic Gene Therapies
8 RHUL - Royal Holloway [University of London]
9 University of Wolverhampton
10 PAnTher - Physiopathologie Animale et bioThérapie du muscle et du système nerveux
11 CHU Pitié-Salpêtrière [AP-HP]
12 UPCité - Université Paris Cité
13 INSERM - Institut National de la Santé et de la Recherche Médicale
14 UF - University of Florida [Gainesville]
2 UMR 1089, Atlantic Gene Therapies
3 Service of Clinical Trials and Databases
4 Neuromuscular Physiology and Evaluation Laboratory
5 Institut de Myologie
6 AIM
7 Atlantic Gene Therapies
8 RHUL - Royal Holloway [University of London]
9 University of Wolverhampton
10 PAnTher - Physiopathologie Animale et bioThérapie du muscle et du système nerveux
11 CHU Pitié-Salpêtrière [AP-HP]
12 UPCité - Université Paris Cité
13 INSERM - Institut National de la Santé et de la Recherche Médicale
14 UF - University of Florida [Gainesville]
Résumé
Duchenne muscular dystrophy (DMD) is an X-linked inherited muscle-wasting disease primarily affecting young boys with a prevalence of 1:5000. The disease is caused by loss-of-function mutations in the gene encoding for the Dystrophin protein and is characterised by systemic, progressive, irreversible and severe loss of muscle function. Among vector systems that allow efficient in vivo gene transfer, recombinant Adeno Associated Virus vectors (rAAV) hold great promise and result in particular in very efficient transduction of skeletal and cardiac muscles.