RE:Brings back memoriesDr Cashman presented on ALS with reference to Sod1. The presentation maybe on the PMN website now.
Title: RACK1 Knockdown Is a Potential Therapeutic Target in ALS and FTLD-TDP
Session: P1: Aging and Dementia: Basic Science (abstract #3494)
Presenter: Neil Cashman, M.D.
Date & Time: April 23, 2023 from 8:00 – 9:00 a.m. ET
ProMIS has evaluated RACK1 as a potential target for ALS and FTLD-TDP. These neurodegenerative disorders are characterized by the formation of pathogenic aggregates of misfolded TAR DNA binding protein 43 (TDP-43) inside neurons which have been observed to co-aggregate with misfolded RACK1, a ribosomal protein. In a cell system, the misfolded form of RACK1 was detected by ProMIS antibodies selective for this RACK1 isoform.
The poster presented describes how RACK1 knockdown was able to reduce TDP-43 aggregation as well as alleviate the TDP-43-induced global suppression of translation in vitro. Knocking down RACK1 also reduced retinal and motor neuron neurodegeneration in D. melanogaster in vivo. These preclinical findings support misfolded RACK1 as a potential therapeutic target for TDP-43 proteinopathy in non-SOD1 and non-FUS ALS as well as FTLD-TDP.
Both poster presentations are available on the Poster andPublications page of the Company’s website at www.promisneurosciences.com.