A New Treatment for Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) Targets G4C2 DNA and RNA Tandem Repeats Using Selective Small Molecules

Technology ID
TT-0120
Description
This project leads ALS and FTD research by targeting G4C2 expansions in C9orf72. Detailed characterization of G4C2 structures and the development of patented molecules that specifically bind to these targets represent a significant step toward a potential cure.
Content
Structural Mastery: Comprehensive mapping of G4C2 DNA and RNA structures associated with ALS and FTD, providing a robust foundation for targeted drug development Innovative Lead Compounds: Groundbreaking identification and development of lead compounds that selectively target pathogenic G4C2 DNA/RNA structures Selective Efficacy: Demonstrated high specificity of small molecules for G4C2 structures, surpassing the selectivity of current anti-G-quadruplex molecules
Inventors
Prof. Guang ZHU
Applicable Industries
Education, Research, and Professional Services
Biomedical, Healthcare and Food
Potential Application
Chemical Synthesis
Drug Delivery
Drug Discovery
Technology Focus
Medical and Biotechnology
TRL
4
Images
Technology image for TT-0120