Did you enjoy the University of Bern's flashMOOC on “RNA – The Future of Medicine” (https://www.youtube.com/watch?v=VYtKSUT90ic) and want to learn more? Then you're in for a treat!
Spinal muscular atrophy (SMA) is a severe genetic disease in which nerve cells in the spinal cord degenerate, leading to muscle weakness and limited mobility. SMA affects approximately 1 in 10,000 newborns.
Adrian Krainer researched RNA splicing and developed Spinraza, the first approved medication for SMA. Spinraza uses an antisense therapy to increase the production of a vital protein, which helps protect the nerve cells.
Adrian Krainer is a biochemist renowned for his pioneering research on RNA splicing. His work led to the development of Spinraza, the first FDA-approved treatment for spinal muscular atrophy (SMA), a genetic disorder causing muscle weakness and atrophy. Krainer holds the St. Giles Foundation Professorship at Cold Spring Harbor Laboratory and has received numerous accolades, including the 2019 Breakthrough Prize in Life Sciences and the 2021 Wolf Prize in Medicine.
