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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Foilsithe in:Gene Ther
Main Authors: Tockner, B, Kocher, T, Hainzl, S, Reichelt, J, Bauer, J W, Koller, U, Murauer, E M
Formáid: Artigo
Teanga:Inglês
Foilsithe: Nature Publishing Group 2016
Ábhair:
Original Article
Rochtain Ar Líne: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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