RNA splicing is a pivotal step of eukaryotic gene expression during which the introns are excised from the precursor (pre-)RNA and the exons are joined together to form mature RNA products (i.e a protein-coding mRNA or long non-coding (lnc)RNAs). The spliceosome, a complex ribonucleoprotein machine, performs pre-RNA splicing with extreme precision. Deregulated splicing is linked to cancer, genetic, and neurodegenerative diseases. Hence, the discovery of small-molecules targeting core spliceosome components represents an appealing therapeutic opportunity.

Role of computational and structural biology in the development of small-molecule modulators of the spliceosome / Rozza, Riccardo; Janoš, Pavel; Spinello, Angelo; Magistrato, Alessandra. - In: EXPERT OPINION ON DRUG DISCOVERY. - ISSN 1746-045X. - 17:10(2022), pp. 1095-1109. [10.1080/17460441.2022.2114452]

Role of computational and structural biology in the development of small-molecule modulators of the spliceosome

Alessandra Magistrato
2022

Abstract

RNA splicing is a pivotal step of eukaryotic gene expression during which the introns are excised from the precursor (pre-)RNA and the exons are joined together to form mature RNA products (i.e a protein-coding mRNA or long non-coding (lnc)RNAs). The spliceosome, a complex ribonucleoprotein machine, performs pre-RNA splicing with extreme precision. Deregulated splicing is linked to cancer, genetic, and neurodegenerative diseases. Hence, the discovery of small-molecules targeting core spliceosome components represents an appealing therapeutic opportunity.
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Rozza, Riccardo; Janoš, Pavel; Spinello, Angelo; Magistrato, Alessandra
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11767/130010
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