Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment for spinal muscular atrophy called Spinraza. The drug has helped over 11,000 patients make more of a protein that certain neurons in the spine need.
A new treatment approach for cystic fibrosis
Antisense oligonucleotides, or ASOs, are molecules that can be used to control protein levels in cells. Cold Spring Harbor Laboratory Professor Adrian Krainer leveraged ASO technology to develop the first FDA-approved treatment for spinal muscular atrophy called Spinraza. The drug has helped over 11,000 patients make more of a protein that certain neurons in the spine need.