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Trans-Splicing Improvement by the Combined Application of Antisense Strategies
Spliceosome-mediated RNA trans-splicing has become an emergent tool for the repair of mutated pre-mRNAs in the treatment of genetic diseases. RNA trans-splicing molecules (RTMs) are designed to induce a specific trans-splicing reaction via a binding domain for a respective target pre-mRNA region. A...
Autores principales: | Koller, Ulrich, Hainzl, Stefan, Kocher, Thomas, Hüttner, Clemens, Klausegger, Alfred, Gruber, Christina, Mayr, Elisabeth, Wally, Verena, Bauer, Johann W., Murauer, Eva M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2015
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4307297/ https://www.ncbi.nlm.nih.gov/pubmed/25569093 http://dx.doi.org/10.3390/ijms16011179 |
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