Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations

RNA trans-splicing has become a versatile tool in the gene therapy of monogenetic diseases. This technique is especially valuable for the correction of mutations in large genes such as COL7A1, which underlie the dystrophic subtype of the skin blistering disease epidermolysis bullosa. Over 800 mutati...

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Bibliographische Detailangaben
Veröffentlicht in:Gene Ther
Hauptverfasser: Tockner, B, Kocher, T, Hainzl, S, Reichelt, J, Bauer, J W, Koller, U, Murauer, E M
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2016
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5097067/
https://ncbi.nlm.nih.gov/pubmed/27434145
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2016.57
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