Therapeutic possibilities for genetic diseases
Exercises
Exercise 1: viral vectors
- methodology
- application
Exercise 2: CRISPR-Cas 9
-
- methodology
- intro
- application
- Leber congenital amaurosis
- Editas
- editas trial
- the science behind Editas work
- what is LCA-IVS26 (in the Intro)
Exercise 3: anti-sense oligos
Exercise 4: Non-viral vectors and mRNA
- methodology
- application