LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice.

doi:10.1038/s41467-023-36186-9

Article

LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice.

Nature communications - 6 Feb 2023

Prakasam Ramachandran, Bonadiman Angela, Andreotti Roberta, Zuccaro Emanuela, Dalfovo Davide, Marchioretti Caterina, Tripathy Debasmita, Petris Gianluca, Anderson Eric N, Migazzi Alice, Tosatto Laura, Cereseto Anna, Battaglioli Elena, Sorarù Gianni, Lim Wooi Fang, Rinaldi Carlo, Sambataro Fabio, Pourshafie Naemeh, Grunseich Christopher, Romanel Alessandro, Pandey Udai Bhan, Contestabile Andrea, Ronzitti Giuseppe, Basso Manuela, Pennuto Maria

Abstract excerpt

Spinobulbar muscular atrophy (SBMA) is caused by CAG expansions in the androgen receptor gene. Androgen binding to polyQ-expanded androgen receptor triggers SBMA through a combination of toxic gain-of-function and loss-of-function mechanisms. Leveraging cell lines, mice, and patient-derived specimens, we show that androgen receptor co-regulators lysine-specific demethylase 1 (LSD1) and protein arginine...

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LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice.

Abstract excerpt

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