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
Hasane Ratni, Martin Ebeling, Renata S. Scalco, Lutz Mueller, Alexander Stephan, 2023. "SMN2 Splicing Modification by Small Molecules – A Blueprint to Tackle the Underlying Genetic Cause of Many Underserved Diseases", The Handbook of Medicinal Chemistry, Simon E Ward, Andrew Davis
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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).