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Proceedings of the National Academy of Sciences - PNAS, ISSN 1091-6490, 2013, Volume 110, Issue 47, pp. E4530 - E4539
Journal Article
Journal Article
The New England journal of medicine, ISSN 1533-4406, 2007, Volume 357, Issue 26, pp. 2677 - 2686
Journal Article
Nucleic acids research, ISSN 1362-4962, 2016, Volume 44, Issue 14, pp. 6549 - 6563
Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the transcript by blocking the RNA-RNA base-pairing or protein-RNA... 
TOXICOLOGY EVALUATION | SYSTEMIC DELIVERY | RESTORES DYSTROPHIN EXPRESSION | SPINAL MUSCULAR-ATROPHY | SURVIVAL MOTOR-NEURON | BUBBLE LIPOSOMES | BIOCHEMISTRY & MOLECULAR BIOLOGY | 2'-O-METHYL PHOSPHOROTHIOATE | PREMESSENGER RNA | SINGLE NUCLEOTIDE | PRE-MESSENGER-RNA | Genetic Therapy | Gene Expression | Animals | Humans | Oligonucleotides, Antisense - therapeutic use | RNA Splicing - genetics | Clinical Trials as Topic | Survey and Summary
Journal Article
The New England journal of medicine, ISSN 1533-4406, 2017, Volume 377, Issue 3, pp. 222 - 232
Journal Article
Development (Cambridge), ISSN 1477-9129, 2008, Volume 135, Issue 10, pp. 1735 - 1743
Journal Article
Neuron (Cambridge, Mass.), ISSN 0896-6273, 2016, Volume 90, Issue 3, pp. 535 - 550
Journal Article