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Session I of CHDI’s therapeutic conference: Seminars by Gill Bates & Judith Frydman

Gill Bates from University College London, described her work on incomplete splicing of the Htt gene, and the implications of these findings for therapeutic development. The splicing product she identified led to the production of a pure exon-1 protein, which in the context of the CAG expansion, is very toxic. Her recent work has been […]