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Article

Amelioration of beta654-thalassemia in mouse model with the knockdown of aberrantly spliced beta-globin mRNA.

Journal of genetics and genomics = Yi chuan xue bao - 1 Oct 2008

Xie Shuyang, Li Wei, Ren Zhaorui, Zhang Jingzhi, Guo Xinbin, Wang Shu, Huang Shuzhen, Zeng Fanyi, Zeng Yi-Tao

Abstract excerpt

Large amounts of aberrantly spliced mRNA from the beta(654) allele was present in erythroid cells, which might impair the erythropoiesis. A therapeutic strategy for beta-thalassemia was explored by knocking down the aberrantly spliced mRNA of beta-globin. Lentiviral vector with siRNA fragment targets on the specific portion of beta(654)-globin aberrantly spliced pre-mRNA was constructed. In HeLa beta(654) cells,...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 18937916
DOI: 10.1016/S1673-8527(08)60080-6

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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