The Handbook of Medicinal Chemistry
Chapter 24: SMN2 Splicing Modification by Small Molecules – A Blueprint to Tackle the Underlying Genetic Cause of Many Underserved Diseases
Published:03 Feb 2023
H. Ratni, M. Ebeling, R. S. Scalco, L. Mueller, and A. Stephan, in The Handbook of Medicinal Chemistry, ed. S. E. Ward and A. Davis, The Royal Society of Chemistry, 2023, ch. 24, pp. 830-846.
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RNA as a drug target offers a wealth of opportunities to therapeutically modulate numerous cellular processes, including those linked to the so-called “undruggable” protein targets. Of particular interest is the modulation of the natural process of pre-mRNA splicing, to control the formation of the corresponding protein products. A successful example of this approach, which will be reviewed here, is the modulation of the SMN2 alternative splicing for the treatment of spinal muscular atrophy (SMA).