Restoration of correct βIVS2-654-globin mRNA splicing and HbA production by engineered U7 snRNA in β-thalassaemia/HbE erythroid cells
Author:
Publisher
Springer Science and Business Media LLC
Subject
Multidisciplinary
Link
http://www.nature.com/articles/s41598-019-43964-3.pdf
Reference29 articles.
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2. Cheng, T. C. et al. beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects. Proc Natl Acad Sci USA 81, 2821–2825 (1984).
3. Hoy, S. M. Nusinersen: First Global Approval. Drugs 77, 473–479 (2017).
4. Syed, Y. Y. Eteplirsen: First Global Approval. Drugs 76, 1699–1704 (2016).
5. Lacerra, G. et al. Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. Proc Natl Acad Sci USA 97, 9591–9596 (2000).
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