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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Bibliografiske detaljer
Udgivet i:Gene Ther
Main Authors: Tockner, B, Kocher, T, Hainzl, S, Reichelt, J, Bauer, J W, Koller, U, Murauer, E M
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
Fag:
Original Article
Online adgang: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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