Selective mTORC1 Inhibitors to Treat TSC
Abstract
mTOR (mammalian target of rapamycin) is a protein kinase that forms two distinct complexes mTORC1 and mTORC2. Aberrant upregulation of mTORC1 signaling has the potential to cause disease, including Tuberous Sclerosis Complex (TSC). We aim to develop novel mTORC1 inhibitors with improved potency and safety over current mTORC1 inhibitors in the market, such as Rapamycin and Everolimus (a rapamycin analog). After initial high throughput screening, we have obtained a class of molecules that inhibit mTORC1 in vitro with a different mode of action than Rapamycin. We are currently optimizing this class of molecules towards activity towards mTORC1 inhibition in cells and in mouse models.
Document Details
- Document Type
- Technical Report
- Publication Date
- May 01, 2023
- Accession Number
- AD1208652
Entities
People
- Dritan Liko
- Michael N. Hall
Organizations
- University of Basel