Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy

PLoS ONE

Volumn 8, Issue 4, 2013, Pages

  Mitrpant, Chalermchai ^a,b   Porensky, Paul ^c   Zhou, Haiyan ^d   Price, Loren ^a   Muntoni, Francesco ^d   Fletcher, Sue ^a   Wilton, Steve D ^a   Burghes, Arthur H M ^c  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^b MAHIDOL UNIVERSITY   (Thailand)

^c THE OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER   (United States)

^d UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BETA ACTIN; MORPHOLINO OLIGONUCLEOTIDE; SURVIVAL MOTOR NEURON PROTEIN 1;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONCENTRATION RESPONSE; CONTROLLED STUDY; DISEASE CARRIER; DISEASE SEVERITY; DOSE RESPONSE; EXON; GENE OVEREXPRESSION; HUMAN; HUMAN CELL; IN VITRO STUDY; INTRON; MOUSE; NEUROPATHOLOGY; NONHUMAN; PHENOTYPE; PROCESS OPTIMIZATION; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN MOTIF; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SEQUENCE ANALYSIS; SPINAL MUSCULAR ATROPHY; SURVIVAL MOTOR NEURON 1 GENE; SURVIVAL RATE; UPREGULATION; WESTERN BLOTTING;

ANIMALS; BASE SEQUENCE; CELLS, CULTURED; GENETIC THERAPY; HUMANS; INJECTIONS, INTRAVENTRICULAR; MICE; MICE, TRANSGENIC; MORPHOLINOS; MUSCULAR ATROPHY, SPINAL; OLIGONUCLEOTIDES, ANTISENSE; RNA INTERFERENCE; RNA PROCESSING, POST-TRANSCRIPTIONAL; RNA, MESSENGER; SURVIVAL OF MOTOR NEURON 2 PROTEIN; TITRIMETRY; TRANSCRIPTION, GENETIC;

EID: 84876444381     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0062114     Document Type: Article

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