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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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| Publicado no: | Sci Rep |
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| Main Authors: | , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4705535/ https://ncbi.nlm.nih.gov/pubmed/26743572 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep18865 |
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