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Therapeutic effects of engineered exosome-based miR-25 and miR-181a treatment in spinocerebellar ataxia type 3 mice by silencing ATXN3

BACKGROUND: Spinocerebellar ataxia type 3 (SCA3) is the most common autosomal dominant hereditary ataxia worldwide, which is however in a lack of effective treatment. In view of that engineered exosomes are a promising non-invasive gene therapy transporter that can overcome the traditional problem o...

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Detalles Bibliográficos
Autores principales: Tang, Zhenchu, Hu, Shenglan, Wu, Ziwei, He, Miao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10337053/
https://www.ncbi.nlm.nih.gov/pubmed/37438701
http://dx.doi.org/10.1186/s10020-023-00695-6