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...

Täydet tiedot

Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Int J Mol Sci
Päätekijät: 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.
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: MDPI 2020
Aiheet:
Article
Linkit: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
Tagit: Lisää tagi
Ei tageja, Lisää ensimmäinen tagi!

Samankaltaisia teoksia