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

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 specimen...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9902531/
https://www.ncbi.nlm.nih.gov/pubmed/36746939
http://dx.doi.org/10.1038/s41467-023-36186-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9902531/
https://www.ncbi.nlm.nih.gov/pubmed/36746939
http://dx.doi.org/10.1038/s41467-023-36186-9

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