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Gene editing for Spinocerebellar ataxia type 3 taking advantage of the human ATXN3L paralog as replacement gene.

Rybarikova M, Rey M, Hasanovic E, Sipion M, Rambousek L, Deglon N
Gene Ther. 2025 Jul 28. doi: 10.1038/s41434-025-00557-2. (Link opens in a new window) PubMed (Link opens in a new window) Article

Plasmids from Article

ID Plasmid Purpose
245084pAAVss-CBA-H2B-mCherry-WPRE-bGHFluorescent reporter gene
245085pAAVss-CBA-H2B-staygold-SYNpAFluorescent reporter gene

Antibodies from Article