Article
TALEN-mediated generation and genetic correction of disease-specific human induced pluripotent stem cells.
Current gene therapy - 1 Jan 2014
Ramalingam Sivaprakash, Annaluru Narayana, Kandavelou Karthikeyan, Chandrasegaran Srinivasan
Abstract excerpt
Generation and precise genetic correction of patient-derived hiPSCs have great potential in regenerative medicine. Such targeted genetic manipulations can now be achieved using gene-editing nucleases. Here, we report generation of cystic fibrosis (CF) and Gaucher's disease (GD) hiPSCs respectively from CF (homozygous for CFTRΔF508 mutation) and Type II GD [homozygous for β-glucocerebrosidase (GBA) 1448T>C...
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