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The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice

β-thalassemia results from point mutations or small deletions in the β-globin (HBB) gene that ultimately cause anemia. The generation of induced pluripotent stem cells (iPSCs) from the somatic cells of patients in combination with subsequent homologous recombination-based gene correction provides ne...

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Detalles Bibliográficos
Autores principales: Ou, Zhanhui, Niu, Xiaohua, He, Wenyin, Chen, Yuchang, Song, Bing, Xian, Yexing, Fan, Di, Tang, Daolin, Sun, Xiaofang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5007518/
https://www.ncbi.nlm.nih.gov/pubmed/27581487
http://dx.doi.org/10.1038/srep32463