Therapeutic development for Canavan disease using patient iPSCs introduced with the wild-type ASPA gene

Canavan disease (CD) is a devastating neurological disease that lacks effective therapy. Because CD is caused by mutations of the aspartoacylase (ASPA) gene, we introduced the wild-type (WT) ASPA gene into patient iPSCs through lentiviral transduction or CRISPR/Cas9-mediated gene editing. We then di...

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Detalles Bibliográficos
Autores principales: Chao, Jianfei, Feng, Lizhao, Ye, Peng, Chen, Xianwei, Cui, Qi, Sun, Guihua, Zhou, Tao, Tian, E, Li, Wendong, Hu, Weidong, Riggs, Arthur D., Matalon, Reuben, Shi, Yanhong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9142666/
https://www.ncbi.nlm.nih.gov/pubmed/35637731
http://dx.doi.org/10.1016/j.isci.2022.104391

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