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Volumn 12, Issue 11, 2013, Pages 813-815
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Make or break for first splice-modulating agents
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NONE
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Author keywords
[No Author keywords available]
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Indexed keywords
ANTISENSE OLIGONUCLEOTIDE;
DRISAPERSEN;
DYSTROPHIN;
ETEPLIRSEN;
MESSENGER RNA PRECURSOR;
PRE MRNA SPLICE MODULATING AGENT;
PROTEIN BCL X;
PROTEIN MCL 1;
SMT C 1100;
SPLICE MODULATING ANTISENSE OLIGONUCLEOTIDE;
UNCLASSIFIED DRUG;
BASE PAIRING;
BETA GLOBIN GENE;
BETA THALASSEMIA;
CLINICAL RESEARCH;
DISEASE COURSE;
DISEASE SEVERITY;
DRUG ACTIVITY;
DRUG APPROVAL;
DRUG BINDING;
DRUG DOSE COMPARISON;
DRUG EFFICACY;
DRUG INDICATION;
DRUG MEGADOSE;
DRUG STRUCTURE;
DUCHENNE MUSCULAR DYSTROPHY;
EXERCISE TEST;
EXERCISE TOLERANCE;
EXON;
FOOD AND DRUG ADMINISTRATION;
GENE;
GENE DELETION;
GENE EXPRESSION REGULATION;
GENE INTERACTION;
HUMAN;
INTRON;
PRIORITY JOURNAL;
PROTEIN EXPRESSION;
PROTEIN FUNCTION;
PROTEIN SYNTHESIS;
RNA SPLICING;
SHORT SURVEY;
SMN1 GENE;
SMN2 GENE;
SPINAL MUSCULAR ATROPHY;
STRUCTURE ANALYSIS;
UTROPHIN GENE;
DRUGS, INVESTIGATIONAL;
HUMANS;
MUSCULAR DYSTROPHY, DUCHENNE;
OLIGONUCLEOTIDES, ANTISENSE;
RNA SPLICING;
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EID: 84887050127
PISSN: 14741776
EISSN: 14741784
Source Type: Journal
DOI: 10.1038/nrd4151 Document Type: Short Survey |
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Times cited : (2)
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References (0)
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