Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy

Annals of the New York Academy of Sciences

Volumn 1175, Issue , 2009, Pages 71-79

  Heemskerk, Hans ^a   De Winter, Christa L ^a   Van Ommen, Gert Jan B ^a   Van Deutekom, Judith C T ^b   Aartsma Rus, Annemieke ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b Prosensa BV   (Netherlands)

Author keywords

Antisense oligonucleotides; Duchenne muscular dystrophy; Exon skipping; Splicing; Therapy

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN;

CLINICAL TRIAL; CONFERENCE PAPER; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXPERIMENTAL MODEL; GENE DELETION; GENE MUTATION; GENE TARGETING; HUMAN; MUSCLE FUNCTION; NONHUMAN; PROTEIN EXPRESSION; TREATMENT DURATION; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

CANIS FAMILIARIS;

EID: 70349451258     PISSN: 00778923     EISSN: 17496632     Source Type: Book Series    
DOI: 10.1111/j.1749-6632.2009.04973.x     Document Type: Conference Paper

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