CRISPR-Mediated In Situ Introduction or Integration of F9-Padua in Human iPSCs for Gene Therapy of Hemophilia B

Hemophilia B (HB) is an X-linked recessive disease caused by F9 gene mutation and functional coagulation factor IX (FIX) deficiency. Patients suffer from chronic arthritis and death threats owing to excessive bleeding. Compared with traditional treatments, gene therapy for HB has obvious advantages,...

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Detalles Bibliográficos
Autores principales: Tang, Qiyu, Hu, Zhiqing, Zhao, Junya, Zhou, Tao, Tang, Shuqing, Wang, Peiyun, Xiao, Rou, Chen, Yan, Wu, Lingqian, Zhou, Miaojin, Liang, Desheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10219373/
https://www.ncbi.nlm.nih.gov/pubmed/37240366
http://dx.doi.org/10.3390/ijms24109013

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