CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia
作者： Mark J. Osborn , Cara-lin Lonetree , Beau R. Webber , Dharmeshkumar Patel , Samantha Dunmire
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摘要: The ability to rationally target disease-causing mutations has been made possible with programmable nucleases the clustered, regularly interspaced short palindromic repeats/Cas9 system representing a facile platform for individualized gene-based medicine. In this study we employed footprint-free reprogramming of fibroblasts from patient Fanconi anemia I (FANCI) gene generate induced pluripotent stem cells (iPSCs). This process was accomplished without complementation and resultant iPSCs were able be corrected in robust manner using Cas9 nickase. self-renewing that maintained under feeder-free conditions differentiated into characteristics definitive hematopoiesis. defined highly efficient procedure small molecule modulation hematopoietic differentiation pathway vascular induction technique progenitors. sum, our results demonstrate induce patien...
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