RNA-Targeted Therapies and High-Throughput Screening Methods.
Int J Mol Sci
; 21(8)2020 Apr 23.
Article
en En
| MEDLINE
| ID: mdl-32340368
ABSTRACT
RNA-binding proteins (RBPs) are involved in regulating all aspects of RNA metabolism, including processing, transport, translation, and degradation. Dysregulation of RNA metabolism is linked to a plethora of diseases, such as cancer, neurodegenerative diseases, and neuromuscular disorders. Recent years have seen a dramatic shift in the knowledge base, with RNA increasingly being recognised as an attractive target for precision medicine therapies. In this article, we are going to review current RNA-targeted therapies. Furthermore, we will scrutinise a range of drug discoveries targeting protein-RNA interactions. In particular, we will focus on the interplay between Lin28 and let-7, splicing regulatory proteins and survival motor neuron (SMN) pre-mRNA, as well as HuR, Musashi, proteins and their RNA targets. We will highlight the mechanisms RBPs utilise to modulate RNA metabolism and discuss current high-throughput screening strategies. This review provides evidence that we are entering a new era of RNA-targeted medicine.
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
ARN
/
Terapia Genética
/
Descubrimiento de Drogas
/
Ensayos Analíticos de Alto Rendimiento
/
Terapia Molecular Dirigida
Tipo de estudio:
Diagnostic_studies
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Screening_studies
Límite:
Animals
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Humans
Idioma:
En
Revista:
Int J Mol Sci
Año:
2020
Tipo del documento:
Article
País de afiliación:
Reino Unido