Drosophila screen connects nuclear transport genes to DPR pathology in c9ALS/FTD - PubMed (original) (raw)

Chimeric Peptide Species Contribute to Divergent Dipeptide Repeat Pathology in c9ALS/FTD and SCA36.

McEachin ZT, Gendron TF, Raj N, García-Murias M, Banerjee A, Purcell RH, Ward PJ, Todd TW, Merritt-Garza ME, Jansen-West K, Hales CM, García-Sobrino T, Quintáns B, Holler CJ, Taylor G, San Millán B, Teijeira S, Yamashita T, Ohkubo R, Boulis NM, Xu C, Wen Z, Streichenberger N; Neuro–CEB Neuropathology Network; Fogel BL, Kukar T, Abe K, Dickson DW, Arias M, Glass JD, Jiang J, Tansey MG, Sobrido MJ, Petrucelli L, Rossoll W, Bassell GJ. McEachin ZT, et al. Neuron. 2020 Jul 22;107(2):292-305.e6. doi: 10.1016/j.neuron.2020.04.011. Epub 2020 May 5. Neuron. 2020. PMID: 32375063 Free PMC article.

C9orf72 ALS/FTD dipeptide repeat protein levels are reduced by small molecules that inhibit PKA or enhance protein degradation.

Licata NV, Cristofani R, Salomonsson S, Wilson KM, Kempthorne L, Vaizoglu D, D'Agostino VG, Pollini D, Loffredo R, Pancher M, Adami V, Bellosta P, Ratti A, Viero G, Quattrone A, Isaacs AM, Poletti A, Provenzani A. Licata NV, et al. EMBO J. 2022 Jan 4;41(1):e105026. doi: 10.15252/embj.2020105026. Epub 2021 Nov 18. EMBO J. 2022. PMID: 34791698 Free PMC article.