Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Nature Medicine

Volumn 21, Issue 3, 2015, Pages 270-275

  Goyenvalle, Aurélie ^a   Griffith, Graziella ^a   Babbs, Arran ^b   Andaloussi, Samir El ^b,c   Ezzat, Kariem ^b   Avril, Aurélie ^a   Dugovic, Branislav ^d   Chaussenot, Rémi ^e,f   Ferry, Arnaud ^g   Voit, Thomas ^g   Amthor, Helge ^a   Bühr, Claudia ^h   Schürch, Stefan ^h   Wood, Matthew J A ^b   Davies, Kay E ^b   Vaillend, Cyrille ^e,f   Leumann, Christian ^h   Garcia, Luis ^a  

^a LABORATOIRE DES SCIENCES DU CLIMAT ET DE L ENVIRONNEMENT   (France)

^b UNIVERSITY OF OXFORD   (United Kingdom)

^c KAROLINSKA INSTITUTE   (Sweden)

^d SYNTHENA AG   (Switzerland)

^e UNIVERSITÉ PARIS SACLAY   (France)

^f CNRS   (France)

^g SORBONNE UNIVERSITÉ   (France)

^h UNIVERSITY OF BERN   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DNA; DYSTROPHIN; NANOPARTICLE; OLIGOMER; TRICYCLO DNA; UNCLASSIFIED DRUG; ANTISENSE OLIGODEOXYNUCLEOTIDE; APO-DYSTROPHIN 1; MESSENGER RNA; STOP CODON; TRANSCRIPTOME;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BICEPS BRACHII MUSCLE; BRAIN; CONTROLLED STUDY; DIAPHRAGM; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GASTROCNEMIUS MUSCLE; GENETIC DISORDER; HEART; HEART FUNCTION; KIDNEY; LIVER; MOUSE; MOUSE MODEL; MUSCLE FUNCTION; MUSCULAR DYSTROPHY; NEUROMUSCULAR DISEASE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; QUADRICEPS FEMORIS MUSCLE; RESPIRATORY FUNCTION; SINGLE DRUG DOSE; SKELETAL MUSCLE; TIBIALIS ANTERIOR MUSCLE; TISSUE DISTRIBUTION; TRICEPS BRACHII MUSCLE; WESTERN BLOTTING; ANIMAL; BLOOD BRAIN BARRIER; DISEASE MODEL; DRUG EFFECTS; EXON; GENE THERAPY; GENETICS; HEART MUSCLE; METABOLISM; STOP CODON; TRANSMISSION ELECTRON MICROSCOPY;

ANIMALS; BLOOD-BRAIN BARRIER; CODON, NONSENSE; DISEASE MODELS, ANIMAL; DYSTROPHIN; EXONS; GENETIC THERAPY; HEART; MICE; MICROSCOPY, ELECTRON, TRANSMISSION; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, DUCHENNE; MYOCARDIUM; NANOPARTICLES; OLIGODEOXYRIBONUCLEOTIDES, ANTISENSE; RNA, MESSENGER; TRANSCRIPTOME;

EID: 84925507915     PISSN: 10788956     EISSN: 1546170X     Source Type: Journal    
DOI: 10.1038/nm.3765     Document Type: Article

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