How RNA-Targeting Medicines Enable Mutation-Specific Treatment

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First session:  January 23rd @ 8AM PST  

   

Sign up here    

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• Monthly course designed for anyone working to bring new hope to patients

• Free software license for managing and developing your own therapeutics

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Agenda: 

• The basics of RNA-targeting therapeutics chemistry

• Real-world strategies used in rare disease drug development

• How to turn harmful genes down

• How turn helpful genes up

• Fix splicing errors that cause disease

• Reduce unwanted side effects through smarter design

• An introduction to siRNA and single stranded antisense therapies

• Clinical examples: SMA, DMD and ALS

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