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Correction of RNA splicing defect in β(654)-thalassemia mice using CRISPR/Cas9 gene-editing technology

β(654)-thalassemia is a prominent Chinese subtype of β-thalassemia, representing 17% of all cases of β-thalassemia in China. The molecular mechanism underlying this subtype involves the IVS-2-654 C→T mutation leading to aberrant β-globin RNA splicing. This results in an additional 73-nucleotide exon...

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Detalles Bibliográficos
Autores principales:	Lu, Dan, Gong, Xiuli, Fang, Yudan, Guo, Xinbing, Chen, Yanwen, Yang, Fan, Zhao, Guijun, Ma, Qingwen, Zeng, Yitao, Zeng, Fanyi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Fondazione Ferrata Storti 2021
Materias:	Article - Red Cell Biology & its Disorders
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9152980/ https://www.ncbi.nlm.nih.gov/pubmed/34706494 http://dx.doi.org/10.3324/haematol.2020.278238

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