In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs

Nearly half of severe Hemophilia A (HA) cases are caused by F8 intron 22 inversion (Inv22). This 0.6-Mb inversion splits the 186-kb F8 into two parts with opposite transcription directions. The inverted 5′ part (141 kb) preserves the first 22 exons that are driven by the intrinsic F8 promoter, leadi...

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Detalles Bibliográficos
Autores principales: Wu, Yong, Hu, Zhiqing, Li, Zhuo, Pang, Jialun, Feng, Mai, Hu, Xuyun, Wang, Xiaolin, Lin-Peng, Siyuan, Liu, Bo, Chen, Fangping, Wu, Lingqian, Liang, Desheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4705535/
https://www.ncbi.nlm.nih.gov/pubmed/26743572
http://dx.doi.org/10.1038/srep18865

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4705535/
https://www.ncbi.nlm.nih.gov/pubmed/26743572
http://dx.doi.org/10.1038/srep18865

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