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Showing 1–3 of 3 results
Advanced filters: Author: Catherine Douthwright Clear advanced filters

  • The GGGGCC repeat expansion in C9ORF72-ALS/FTD can be translated into five dipeptide repeat (DPR) proteins, including poly(GR) and poly(GA). Here, the authors develop assays to detect the levels of these DPR proteins in the CSF of individuals with ALS/FTD.

    • Gopinath Krishnan
    • Denitza Raitcheva
    • Fen-Biao Gao
    ResearchOpen Access
    Nature Communications
    Volume: 13, P: 1-8

  • First-in-human combined intrathalamic and intrathecal gene therapy in two patients with Tay-Sachs disease provides early evidence on the safety and feasibility of the approach.

    • Terence R. Flotte
    • Oguz Cataltepe
    • Miguel Sena-Esteves
    Research
    Nature Medicine
    Volume: 28, P: 251-259