RE:RE:Brings back memoriesSummary from dr. Cashman preaentation
Summary
• ProMIS monoclonal antibodies selective for misfolded RACK1 (RACK1mis mAb) showed coaggregation of RACK1 and TDP-43 in ALS and FTLD-TDP, and in cell culture;
• RACK1 knockdown alleviated TDP-43 aggregation as well as associated global translational suppression in cultured cells;
• RACK1 knockdown was well-tolerated in cultured cells in vitro and D. melanogaster neurons in vivo;
• RACK1 knockdown ameliorated retinal and motor neuron neurodegeneration in D. melanogaster in vivo;
• These findings support RACK1 knockdown as a potential therapeutic approach for TDP-43 proteinopathy in non-SOD1