Thymosin α1 represents a potential potent single-molecule-based therapy for cystic fibrosis
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Biochemistry, Genetics and Molecular Biology,General Medicine
Link
http://www.nature.com/articles/nm.4305.pdf
Reference69 articles.
1. Rowe, S.M., Miller, S. & Sorscher, E.J. Cystic fibrosis. N. Engl. J. Med. 352, 1992–2001 (2005).
2. Lukacs, G.L. & Verkman, A.S. CFTR: folding, misfolding and correcting the ΔF508 conformational defect. Trends Mol. Med. 18, 81–91 (2012).
3. Okiyoneda, T. et al. Peripheral protein quality control removes unfolded CFTR from the plasma membrane. Science 329, 805–810 (2010).
4. Pedemonte, N. et al. Small-molecule correctors of defective ΔF508-CFTR cellular processing identified by high-throughput screening. J. Clin. Invest. 115, 2564–2571 (2005).
5. Galietta, L.J. Managing the underlying cause of cystic fibrosis: a future role for potentiators and correctors. Paediatr. Drugs 15, 393–402 (2013).
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