Efficient gene correction of an aberrant splice site in β‐thalassaemia iPSCs by CRISPR/Cas9 and single‐strand oligodeoxynucleotides

β‐thalassaemia is a prevalent hereditary haematological disease caused by mutations in the human haemoglobin β (HBB) gene. Among them, the HBB IVS2‐654 (C > T) mutation, which is in the intron, creates an aberrant splicing site. Bone marrow transplantation for curing β‐thalassaemia is limited due...

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Detalles Bibliográficos
Autores principales: Xiong, Zeyu, Xie, Yingjun, Yang, Yi, Xue, Yanting, Wang, Ding, Lin, Shouheng, Chen, Diyu, Lu, Dian, He, Lina, Song, Bing, Yang, Yinghong, Sun, Xiaofang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6850948/
https://www.ncbi.nlm.nih.gov/pubmed/31631510
http://dx.doi.org/10.1111/jcmm.14669

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