About the Program
A low molecular weight compound that is small enough to easily get into tissues, enter cells, and interact with specific biological targets. In AS, small molecules could be developed that function as drugs by modulating biochemical pathways, inhibiting or activating specific proteins, or altering cellular processes shown to be altered in AS neurons. These targets of small molecules can be proteins, DNA, RNA or the ATS.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A