Rubika collaborated with the Ule lab to identify the RNA transcripts bound by C9orf72 polyPR in cells. We hope this will be a useful resource for the field, all the data is freely available: https://www.life-science-alliance.org/content/6/9/e202201824 With co-first author Igor Ruiz de los Mozos they identified the GAAGA pentamer to be…
Ashling was selected to give a talk at the GRS on ALS in Les Diablerets in Switzerland in July 2023. She presented her exciting data on neuronal lipid homeostasis in C9orf72 FTD/ALS.
Alex gave a short talk at the MNDA EnCouRage meeting in June 2023, an MND patient-facing meeting for ECRs hosted by the MNDA. Thanks to the MNDA for putting on this insightful event. https://www.mndassociation.org/research/research/mnd-encourage-uk-2023
George gave a brilliant flash talk and poster on identifying new regulators of plasma and lysosomal membrane repair with Jez Carlton’s lab – great work George!
Christina will be working on a gene therapy project for ALS/FTD. We are proud supporters of the In2research Programme: https://in2scienceuk.org/in2research/
We developed an ultra-sensitive Simoa-based immunoassay for detecting C9orf72 polyGP in CSF. In collaboration with Wave Life Sciences we validated the assay for clinical trial use and validated it using samples from the Genetic FTD Initiative. A great collaboration with Henrik Zetterberg, Amanda Heslegrave, Jon Rohrer and Wave.
A nice collaboration with Alessandro Provenzani’s lab on small molecule regulators of C9orf72 RAN translation. Sally investigated their effect in iPSC-motor neurons derived from people with C9orf72 repeat expansions. Paper just out in EMBO Journal: https://www.embopress.org/doi/full/10.15252/embj.2020105026