The spliceosome: a novel multi-faceted target for therapy

Tazi, J.; Durand, S.; Jeanteur, P.

Trends in biochemical sciences

2005-08 / vol 30 / pages 469-78


The spliceosome is a dynamic and flexible ribonucleoprotein enzyme that removes intronic sequences in a regulated manner. Spliceosome action enables one stretch of genomic DNA sequence to yield several mRNAs that encode different proteins. It depends on a flexible mechanism for selecting splice sites, which calls for regulatory sequences (splicing enhancers or silencers) recognized by cognate trans-acting protein factors and constitutive ribonucleoprotein devices to build up the catalytic core. The identification of both types of elements now offers a comprehensive insight into how the spliceosome is adapted to carry out the removal of different introns and suggests novel therapeutic targets to, ultimately, restore a physiological pattern of alternatively spliced variants in a large repertoire of pathologies.

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Humans; Animals; Phosphorylation; RNA Splicing/genetics; Introns/genetics; *Drug Design; Adenosine Triphosphate/metabolism; Spliceosomes/*drug effects/*metabolism

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