Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia-Reference-Cited by-同舟云学术

Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia

Published:2017-09-04 Issue:1 Volume:8 Page:
ISSN:2041-1723
Container-title:Nature Communications
language:en
Short-container-title:Nat Commun

Author:

Mettananda Sachith^ORCID,Fisher Chris A.,Hay Deborah,Badat Mohsin,Quek Lynn,Clark Kevin,Hublitz Philip,Downes Damien,Kerry Jon,Gosden Matthew,Telenius Jelena,Sloane-Stanley Jackie A.,Faustino Paula,Coelho Andreia,Doondeea Jessica,Usukhbayar Batchimeg,Sopp Paul,Sharpe Jacqueline A.,Hughes Jim R.^ORCID,Vyas Paresh^ORCID,Gibbons Richard J.,Higgs Douglas R.^ORCID

Publisher

Springer Science and Business Media LLC

Subject

General Physics and Astronomy,General Biochemistry, Genetics and Molecular Biology,General Chemistry

Link

http://www.nature.com/articles/s41467-017-00479-7.pdf

Reference35 articles.

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2. Weatherall D. J. & Clegg J. B. in The Thalassaemia Syndromes, 4 edn (Blackwell Science, 2001).

3. Mettananda, S., Gibbons, R. J. & Higgs, D. R. Alpha-Globin as a molecular target in the treatment of beta-thalassemia. Blood 125, 3694–3701 (2015).

4. Sollaino, M. C. et al. Association of alpha globin gene quadruplication and heterozygous beta thalassemia in patients with thalassemia intermedia. Haematologica 94, 1445–1448 (2009).

5. Premawardhena, A. et al. A novel molecular basis for beta thalassemia intermedia poses new questions about its pathophysiology. Blood 106, 3251–3255 (2005).

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