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...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:Int J Mol Sci
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2020
Témata:
Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky