A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta‐Thalassemia and Sickle Cell Disease

Beta‐thalassemia is one of the most common recessive genetic diseases, caused by mutations in the HBB gene. Over 200 different types of mutations in the HBB gene containing three exons have been identified in patients with β‐thalassemia (β‐thal) whereas a homozygous mutation in exon 1 causes sickle...

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Detalles Bibliográficos
Autores principales: Cai, Liuhong, Bai, Hao, Mahairaki, Vasiliki, Gao, Yongxing, He, Chaoxia, Wen, Yanfei, Jin, You‐Chuan, Wang, You, Pan, Rachel L., Qasba, Armaan, Ye, Zhaohui, Cheng, Linzhao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Translational Research Articles and Reviews
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5746148/
https://www.ncbi.nlm.nih.gov/pubmed/29164808
http://dx.doi.org/10.1002/sctm.17-0066

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5746148/
https://www.ncbi.nlm.nih.gov/pubmed/29164808
http://dx.doi.org/10.1002/sctm.17-0066

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