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Designing Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair

RNA trans-splicing is a promising tool for mRNA modification in a diversity of genetic disorders. In particular, the substitution of internal exons of a gene by combining 3′ and 5′ RNA trans-splicing seems to be an elegant way to modify especially large pre-mRNAs. Here we discuss a robust method for...

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Detalles Bibliográficos
Autores principales: Hüttner, Clemens, Murauer, Eva M., Hainzl, Stefan, Kocher, Thomas, Neumayer, Anna, Reichelt, Julia, Bauer, Johann W., Koller, Ulrich
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5085642/
https://www.ncbi.nlm.nih.gov/pubmed/27669223
http://dx.doi.org/10.3390/ijms17101609