Lesson 12 – Factors that may affect PS ASO pharmacological effects
Lessons in Antisense [...]
Lesson 11 – Receptor Theory Adapted for RNA-targeted Drugs
Lessons in Antisense [...]
Commitment to Safety First: The DSMB
with Eugene Schneider, M.D.
Lesson 10 – RNase H1
Lessons in Antisense [...]
Lesson 9 – ASO Pharmacodynamic Principles (Part 2)
Lessons in Antisense [...]
Connor’s Story: A First for TUBB4A Treatment
with Diana and Mike Gooley
Lesson 8 – ASO Pharmacodynamic Principles (Part 1)
Lessons in Antisense [...]
Lesson 7 – Peak Plasma PS ASO Concentration-related Toxicities
Lessons in Antisense [...]
What Are iPSCs—and Why Do They Matter?
Induced [...]
Lesson 6 – “Targeting” Ligand Approaches
Lessons in Antisense [...]
