Translational and regulatory challenges for exon skipping therapies

Human Gene Therapy

Volumn 25, Issue 10, 2014, Pages 885-892

  Aartsma Rus, Annemieke ^a,b   Ferlini, Alessandra ^c   Goemans, Nathalie ^d   Pasmooij, Anna M G ^e,f   Wells, Dominic J ^g   Bushby, Katerine ^b   Vroom, Elizabeth ^h   Balabanov, Pavel ⁱ  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b NEWCASTLE UNIVERSITY   (United Kingdom)

^c UNIVERSITY OF FERRARA   (Italy)

^d UNIVERSITY HOSPITALS LEUVEN   (Belgium)

^e MEDICINES EVALUATION BOARD   (Netherlands)

^f UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

^g ROYAL VETERINARY COLLEGE   (United Kingdom)

^h Duchenne Parent Project   (Netherlands)

ⁱ EUROPEAN MEDICINES AGENCY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DNA; RNA;

ANTISENSE THERAPY; CONFERENCE PAPER; DNA SPLICING; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENE MUTATION; HUMAN; NONHUMAN; OUTCOME ASSESSMENT; RARE DISEASE; RNA SPLICING; TRANSLATION REGULATION; WORKSHOP; ANIMAL; EXON; GENE THERAPY; GENETICS; LEGISLATION AND JURISPRUDENCE; MUSCULAR DYSTROPHY, DUCHENNE; NETHERLANDS; PROCEDURES;

ANIMALS; EXONS; GENETIC THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; NETHERLANDS; OLIGONUCLEOTIDES, ANTISENSE; RNA SPLICING;

EID: 84907470441     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.086     Document Type: Conference Paper

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