A new tool is now available to ALS researchers worldwide. The monoclonal antibody, developed by Target ALS in New York, detects the dipeptide repeat protein poly-GP. The approach may help researchers create therapies for C9orf72 ALS by assessing target engagement (see April 2017 news).
The strategy is based on a sandwich ELISA assay, introduced by Tania Gendron, Leonard Petrucelli and colleagues at the Mayo Clinic in Jacksonville, Florida, which measures the levels of poly-GP in the CSF (see April 2017 news; Su et al., 2014). The assay is emerging as a potentially key strategy to evaluate RNA-targeted therapies being developed for C9orf72 ALS due to its ability to indirectly measure the impact of these potential treatments on the levels of repeat-rich C9orf72 RNAs (see March 2017 news; Gendron et al., 2017).
Efforts to create potential treatments for C9orf72 ALS are underway (see October 2017 news). One strategy, being developed by Wave Life Sciences in Cambridge, Massachusetts in collaboration with Robert Brown and colleagues at University of Massachusetts Medical School, aims to protect motor neurons in ALS by reducing levels of repeat-rich C9orf72 RNAs with antisense oligonucleotides (see May 2018 conference news). A clinical trial is anticipated to begin as early as the end of 2018.
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