Context base editing for splice correction of IVSI-110 β-thalassemia
Author:
Funder
European Regional Development Fund
Publisher
Elsevier BV
Reference50 articles.
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3. β+ Thalassemia: Aberrant splicing results from a single point mutation in an intron;Busslinger;Cell,1981
4. Relative and absolute quantification of aberrant and normal splice variants in hbbivsi−110 (G > a) β-thalassemia;Patsali;Int. J. Mol. Sci.,2020
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Cited by 2 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献
1. Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β654-Thalassemia Mouse Model;Human Gene Therapy;2024-08-13
2. Precision genome editing offers hope for treatment of β-thalassemia and other genetic disorders;Molecular Therapy - Nucleic Acids;2024-06
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