Correction of human phospholamban R14del mutation associated with cardiomyopathy using targeted nucleases and combination therapy
Author:
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/ncomms7955.pdf
Reference50 articles.
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2. Dellefave, L. & McNally, E. M. The genetics of dilated cardiomyopathy. Curr. Opin. Cardiol. 25, 198–204 (2010).
3. Schmitt, J. P. et al. Dilated cardiomyopathy and heart failure caused by a mutation in phospholamban. Science 299, 1410–1413 (2003).
4. Haghighi, K. et al. Human phospholamban null results in lethal dilated cardiomyopathy revealing a critical difference between mouse and human. J. Clin. Invest. 111, 869–876 (2003).
5. Haghighi, K. et al. A mutation in the human phospholamban gene, deleting arginine 14, results in lethal, hereditary cardiomyopathy. Proc. Natl Acad. Sci. USA 103, 1388–1393 (2006).
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