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Journal Article
Nucleic acids research, ISSN 0305-1048, 2017, Volume 45, Issue 21, pp. 12214 - 12240
...) gene coupled with predominant skipping of SMN2 exon 7. The only approved SMA treatment is an antisense oligonucleotide that targets the intronic splicing silencer N1 (ISS-N1... 
DISTANCE | U1 SNRNP | SURVIVAL MOTOR-NEURON | SMALL NUCLEAR-RNA | BIOCHEMISTRY & MOLECULAR BIOLOGY | INTRONIC STRUCTURE | SELECTION | CRITICAL EXON | SMN GENE | SINGLE NUCLEOTIDE | PRE-MESSENGER-RNA | Gene regulation, Chromatin and Epigenetics
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