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Updated: Nov 1, 2025

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
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Risdiplam, the First Approved Small Molecule Splicing Modifier Drug as a Blueprint for Future Transformative

Hasane Ratni1, Renata S Scalco1, Alexander H Stephan1

  • 1pRED, Pharma Research & Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd., Grenzacherstrasse 124, 4070 Basel, Switzerland.

ACS Medicinal Chemistry Letters
|June 18, 2021
PubMed

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