Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

Disease-causing splicing mutations can be rescued by variants of the U1 small nuclear RNA (U1snRNAs). However, the evaluation of the efficacy and safety of modified U1snRNAs as therapeutic tools is limited by the availability of cellular and animal models specific for a given mutation. Hence, we exp...

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Bibliographic Details
Published in:Mol Ther Nucleic Acids
Main Authors: Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2016
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5155329/
https://ncbi.nlm.nih.gov/pubmed/27898092
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.97
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