Gene therapy strategies in the treatment of hypertrophic cardiomyopathy
Author:
Funder
DZHK
Publisher
Springer Science and Business Media LLC
Subject
Physiology (medical),Clinical Biochemistry,Physiology
Link
http://link.springer.com/content/pdf/10.1007/s00424-018-2173-5.pdf
Reference79 articles.
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2. Amoasii L, Long C, Li H, Mireault AA, Shelton JM, Sanchez-Ortiz E, McAnally JR, Bhattacharyya S, Schmidt F, Grimm D, Hauschka SD, Bassel-Duby R, Olson EN (2017) Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. Sci Transl Med 9:eaan8081
3. Ashrafian H, Watkins H (2007) Reviews of translational medicine and genomics in cardiovascular disease: new disease taxonomy and therapeutic implications cardiomyopathies: therapeutics based on molecular phenotype. J Am Coll Cardiol 49:1251–1264
4. Azibani F, Brull A, Arandel L, Beuvin M, Nelson I, Jollet A, Ziat E, Prudhon B, Benkhelifa-Ziyyat S, Bitoun M, Lorain S, Bonne G, Bertrand AT (2018) Gene therapy via trans-splicing for LMNA-related congenital muscular dystrophy. Mol Ther Nucleic Acids 10:376–386
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