A collaboration between the University of Pittsburgh and the University of Pennsylvania has led to the development of bait oligonucleotides that inhibit and reverse TDP-43 inclusions associated with ALS, FTD, and Alzheimer's Disease.
Description
These chemically modified oligonucleotides have shown efficacy in preventing and reversing TDP-43 aggregation in vitro, restoring TDP-43 function in iPSC neuronal models, and preventing neuronal loss in mouse models.
Applications
Effective in multiple models, chemical modifications, and broad therapeutic potential.
Invention Readiness
These chemically modified oligonucleotides have shown efficacy in preventing and reversing TDP-43 aggregation in vitro, restoring TDP-43 function in iPSC neuronal models, and preventing neuronal loss in mouse models.
IP Status
Research Tool
