Supramolecular nanosubstrate–mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies

Leveraging the endogenous homology-directed repair (HDR) pathway, the CRISPR-Cas9 gene-editing system can be applied to knock in a therapeutic gene at a designated site in the genome, offering a general therapeutic solution for treating genetic diseases such as hemoglobinopathies. Here, a combined s...

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Publicat a:Sci Adv
Autors principals: Yang, Peng, Chou, Shih-Jie, Li, Jindian, Hui, Wenqiao, Liu, Wenfei, Sun, Na, Zhang, Ryan Y., Zhu, Yazhen, Tsai, Ming-Long, Lai, Henkie I., Smalley, Matthew, Zhang, Xinyue, Chen, Jiayuan, Romero, Zulema, Liu, Dahai, Ke, Zunfu, Zou, Chang, Lee, Chin-Fa, Jonas, Steven J., Ban, Qian, Weiss, Paul S., Kohn, Donald B., Chen, Kai, Chiou, Shih-Hwa, Tseng, Hsian-Rong
Format: Artigo
Idioma:Inglês
Publicat: American Association for the Advancement of Science 2020
Matèries:
Research Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7608838/
https://ncbi.nlm.nih.gov/pubmed/33097539
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.abb7107
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