Citations 2
CRISPR/Cas9-mediated introduction of the sodium/iodide symporter gene enables noninvasive in vivo tracking of induced pluripotent stem cell-derived cardiomyocytes
Ostrominski, John W. , Yada, Ravi Chandra , Sato, Noriko , Klein, Michael G. , Blinova, Ksenia , Valadez, Racquel, Palisoc, Maryknoll, Peng, Kah-Whye , San, Hong, Lin, Yongshun, Basuli, Falguni , Swenson, Rolf E. , Haigney, Mark C. , Boehm, Manfred , Hong, So Gun , Hong, So Gun , Dunbar, Cynthia E , Patel, Dakshesh , Pittaluga, Stefania , Zhang, Xiang , Choyke, Peter L. , Zou, Jizhong
Stem Cells Translational Medicine | July 23, 2020 | Volume: 9 | Issue: 10 | Pages: 1203-1217
Abstract Techniques that enable longitudinal tracking of cell fate after myocardial delivery are imperative for optimizing the efficacy of cell-based cardiac therapies. However, these approaches have been underutilized in preclinical models and clinical trials, and there is considerable demand for site-specific strategies achieving long-term expression of reporter genes compatible with safe noninvasive imaging. In this study, the rhesus sodium/iodide symporter (NIS) gene was incorporated into rhesus macaque induced pluripotent stem cells (RhiPSCs) via CRISPR/Cas9. Cardiomyocytes derived from NIS-RhiPSCs (NIS-RhiPSC-CMs) exhibited overall similar morphological and electrophysiological characteristics compared to parental control RhiPSC-CMs at baseline and with exposure to physiological levels of sodium iodide. Mice were injected intramyocardially with 2 million NIS-RhiPSC-CMs immediately following myocardial infarction, and serial positron emission tomography/computed tomography was performed with 18F-tetrafluoroborate to monitor transplanted cells in vivo. NIS-RhiPSC-CMs could be detected until study conclusion at 8 to 10 weeks postinjection. This NIS-based molecular imaging platform, with optimal safety and sensitivity characteristics, is primed for translation into large-animal preclinical models and clinical trials.
Gene Transfer to Arteries
Akyürek, Levent M. , San, Hong, Nabel, Elizabeth G., Singh, Ripudamanjit, Simari, Robert D.
Current Protocols in Human Genetics | June 9, 2004 | Volume: 31 | Issue: 1 | Pages: No page available
AbstractThis unit provides a set of protocols for introducing recombinant genes into normal, injured, and atherosclerotic arteries. The protocols include animal preparation, surgical techniques, and delivery systems. Protocols describe gene delivery to normal, injured, and stented porcine iliofemoral arteries, employing a double balloon infusion catheter to deliver the vector. Another basic protocol describes gene delivery to atherosclerotic arteries using a hyperlipidemic double‐injury rabbit model, and requires surgical exposure of the artery and instillation of the gene vector via a catheter. Additional protocols describe gene delivery to normal and injured murine carotid and femoral arteries. An describes a percutaneous method for arterial gene delivery. These protocols may be adapted to deliver genes to either injured or noninjured atherosclerotic arteries.