Restoration of GBE1 Protein Activity with an ASO: Impact of a Common Sequencing Error on Drug Discovery
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Lesson 17 – The Science of ASO Design and Screening Using the FDA Guidance for Nano-rare Patients
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Lesson 16 – PS ASO-induced PS ASO-RNP Aggregates
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Lesson 15 – The Mechanisms of PS ASO-induced Cytotoxicity
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Lesson 14 – PS ASO Cellular Uptake and Distribution
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Lesson 13 – PS ASOs and Innate Immunity
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Lesson 12 – Factors that may affect PS ASO pharmacological effects
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Lesson 11 – Receptor Theory Adapted for RNA-targeted Drugs
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Lesson 10 – RNase H1
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Lesson 9 – ASO Pharmacodynamic Principles (Part 2)
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