Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes
Matsa, Elena and Dixon, James E. and Medway, Christopher and Georgiou, Orestis and Patel, Minal J. and Morgan, Kevin and Kemp, Paul J. and Staniforth, Andrew and Mellor, Ian and Denning, Chris (2013) Allele-specific RNA interference rescues the long-QT syndrome phenotype in human-induced pluripotency stem cell cardiomyocytes. European Heart Journal . ISSN 0195-668X
Aims Long-QT syndromes (LQTS) are mostly autosomal-dominant congenital disorders associated with a 1:1000 mutation frequency, cardiac arrest, and sudden death. We sought to use cardiomyocytes derived from human-induced pluripotency stem cells (hiPSCs) as an in vitro model to develop and evaluate gene-based therapeutics for the treatment of LQTS.
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