Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Nature Communications

Volumn 8, Issue , 2017, Pages

  Le Guiner, Caroline ^a,b   Servais, Laurent ^c   Montus, Marie ^b   Larcher, Thibaut ^d   Fraysse, Bodvaël ^a   Moullec, Sophie ^d   Allais, Marine ^a   François, Virginie ^a   Dutilleul, Maeva ^d   Malerba, Alberto ^e   Koo, Taeyoung ^e   Thibaut, Jean Laurent ^f,g   Matot, Béatrice ^f   Devaux, Marie ^a   Le Duff, Johanne ^a   Deschamps, Jack Yves ^d   Barthelemy, Inès ^g,h   Blot, Stéphane ^g,h   Testault, Isabelle ⁱ   Wahbi, Karim ^j   Ederhy, Stéphane ^k   Martin, Samia ^b   Veron, Philippe ^b   Georger, Christophe ^b   Athanasopoulos, Takis ^e,l,p   Masurier, Carole ^b   Mingozzi, Federico ^b   Carlier, Pierre ^f   Gjata, Bernard ^b   Hogrel, Jean Yves ^m   Adjali, Oumeya ^a   Mavilio, Fulvio ^b   Voit, Thomas ⁿ   Moullier, Philippe ^a,o   Dickson, George ^e   more..

^a UNIVERSITÉ DE NANTES   (France)

^b GÉNÉTHON   (France)

^c ARMAND TROUSSEAU HOSPITAL   (France)

^d LUNAM UNIVERSITÉ   (France)

^e UNIVERSITY OF LONDON   (United Kingdom)

^f DIF   (France)

^g UNIVERSITÉ PARIS EST   (France)

^h université Paris Est Créteil   (France)

ⁱ Centre Hospitalier Vétérinaire Atlantia   (France)

^j Reference Center for Muscle Diseases ^*  (France)

^k HÔPITAL SAINT ANTOINE   (France)

^l UNIVERSITY OF WOLVERHAMPTON   (United Kingdom)

^m Institut de Myologie   (France)

ⁿ GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^o UNIVERSITY OF FLORIDA   (United States)

^p GLAXOSMITHKLINE   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; GAMMA INTERFERON; MICRODYSTROPHIN; UNCLASSIFIED DRUG;

CANID; CHRONIC WASTING DISEASE; DISEASE VECTOR; GENE EXPRESSION; LIMB; MUTATION; TOXICITY; VIRUS;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY LABELING; ARTICLE; CANINE MODEL; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; ENZYME LINKED IMMUNOSPOT ASSAY; FOLLOW UP; FORELIMB; GOLDEN RETRIEVER; HISTOLOGY; HISTOPATHOLOGY; IMMUNE RESPONSE; IMMUNOHISTOCHEMISTRY; IMMUNOPEROXIDASE STAINING; MALE; MUSCLE BIOPSY; MUSCLE STRENGTH; NONHUMAN; NUCLEAR MAGNETIC RESONANCE; PERIPHERAL BLOOD MONONUCLEAR CELL; PHENOTYPE; PHOSPHORUS NUCLEAR MAGNETIC RESONANCE; PROMOTER REGION; PROTON NUCLEAR MAGNETIC RESONANCE; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SKELETAL MUSCLE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WESTERN BLOTTING; ANIMAL; ANIMAL MUSCULAR DYSTROPHY; DEPENDOPARVOVIRUS; DISEASE MODEL; DOG; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; INTRAVENOUS DRUG ADMINISTRATION; METABOLISM; PATHOPHYSIOLOGY; TRANSGENE;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS;

ADMINISTRATION, INTRAVENOUS; ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DOGS; DYSTROPHIN; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; MALE; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; MUSCULAR DYSTROPHY, DUCHENNE; TRANSGENES;

EID: 85031709047     PISSN: None     EISSN: 20411723     Source Type: Journal    
DOI: 10.1038/ncomms16105     Document Type: Article

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