A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I

The elucidation of aberrant splicing mechanisms, frequently associated with disease has led to the development of RNA therapeutics based on the U1snRNA, which is involved in 5′ splice site (5′ss) recognition. Studies in cellular models have demonstrated that engineered U1snRNAs can rescue different...

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Publicado en:Int J Mol Sci
Main Authors: Balestra, Dario, Scalet, Daniela, Ferrarese, Mattia, Lombardi, Silvia, Ziliotto, Nicole, C. Croes, Chrystal, Petersen, Naomi, Bosma, Piter, Riccardi, Federico, Pagani, Franco, Pinotti, Mirko, van de Graaf, Stan F. J.
Formato: Artigo
Idioma:Inglês
Publicado: MDPI 2020
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7139742/
https://ncbi.nlm.nih.gov/pubmed/32244944
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21062136
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