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The Handbook of Medicinal Chemistry
Edited by
Andrew Davis
Andrew Davis
AstraZeneca, Sweden
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Hardback ISBN:
978-1-78801-898-2
PDF ISBN:
978-1-83767-107-6
EPUB ISBN:
978-1-83916-588-7
No. of Pages:
994
Publication date:
03 Feb 2023
Book Chapter
Chapter 24: SMN2 Splicing Modification by Small Molecules – A Blueprint to Tackle the Underlying Genetic Cause of Many Underserved Diseases
By
Hasane Ratni
;
Hasane Ratni
F. Hoffmann-La Roche Ltd, pRED, Pharma Research & Early Development, Roche Innovation Center Basel
Grenzacherstrasse 124
4070 Basel
Switzerland
ratnih@yahoo.fr
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Martin Ebeling
;
Martin Ebeling
F. Hoffmann-La Roche Ltd, pRED, Pharma Research & Early Development, Roche Innovation Center Basel
Grenzacherstrasse 124
4070 Basel
Switzerland
ratnih@yahoo.fr
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Renata S. Scalco
;
Renata S. Scalco
F. Hoffmann-La Roche Ltd, pRED, Pharma Research & Early Development, Roche Innovation Center Basel
Grenzacherstrasse 124
4070 Basel
Switzerland
ratnih@yahoo.fr
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Lutz Mueller
;
Lutz Mueller
F. Hoffmann-La Roche Ltd, pRED, Pharma Research & Early Development, Roche Innovation Center Basel
Grenzacherstrasse 124
4070 Basel
Switzerland
ratnih@yahoo.fr
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Alexander Stephan
Alexander Stephan
F. Hoffmann-La Roche Ltd, pRED, Pharma Research & Early Development, Roche Innovation Center Basel
Grenzacherstrasse 124
4070 Basel
Switzerland
ratnih@yahoo.fr
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Published:03 Feb 2023
Page range:
830 - 846
Citation
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).
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