MeiraGTx Targeting TDP-43 Toxicity in Gene Therapy Program

Gene therapy startup company MeiraGTx has announced expansion of its gene therapy program targeting ALS, in collaboration with Gregory Petsko of Weill Cornell Medical College in New York and Ronald Klein of Louisiana State University Health Sciences Center in Shreveport. The company’s gene therapy program is targeting the upframeshift protein 1 (UPF1), a master regulator of the nonsense-mediated mRNA decay (NMD) pathway, which disposes of mRNAs with premature termination codons. In animal models expressing TDP-43, overexpression of UPF1 preserved motor function as compared to untreated controls (see June 2015 news; Jackson et al., 2015). Interestingly, UPF1 has also been shown to be a suppressor of FUS toxicity in cellular models (Barmada et al., 2015), offering an additional direction for applying this candidate therapy. The partners have entered into a research collaboration for pre-clinical testing of this therapy in animal models.

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disease-als disease-ftd FUS tdp-43 topic-preclinical topic-randd upf1
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