Development of Engineered-U1 snRNA Therapies: Current Status

Development of Engineered-U1 snRNA Therapies: Current Status

Splicing of pre-mRNA is a crucial regulatory stage in the pathway of gene expression. The majority of human genes that encode proteins undergo alternative pre-mRNA splicing and mutations that affect splicing are more prevalent than previously thought. Targeting aberrant RNA(s) may thus provide an op...

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Bibliographic Details
Main Authors: Mariana Gonçalves, Juliana Inês Santos, Maria Francisca Coutinho, Liliana Matos, Sandra Alves
Format: Article
Language:English
Published: MDPI AG 2023-09-01
Series:International Journal of Molecular Sciences
Subjects:
U1 snRNA (small nuclear RNA)-based therapies
modified U1 snRNAs
exon-specific U1 snRNAs (ExSpeU1s)
5′ splice-site (5′ss)
splicing mutations
splicing correction
Online Access:https://www.mdpi.com/1422-0067/24/19/14617

Internet

https://www.mdpi.com/1422-0067/24/19/14617

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