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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Pubblicato in:Int J Mol Sci
Autori principali: 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.
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2020
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Article
Accesso online: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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