AUG 30, 2016 8:00 AM PDT

Delivery solutions for robust CRISPR-based genomic editing in stem cells for disease model generation


Stem cells, specifically induced pluripotent stem cells (iPSCs), offer exciting potential for the future of cell therapy and regenerative medicine. More recently, genetic engineering of stem cells and progenitor lines is enabling in vitro disease modeling for drug development with cell populations that would otherwise be unavailable from live donors.  With the use of TALENs and CRISPRs, this true power is fast approaching.  Having the ability to develop two cell models with isogenic background, except for the site specific edit, gives researchers the potential to study the true effects of a single mutation in a pathway or syndrome.  However, the lack of advanced technologies has been hindering the current pace of research and discovery.  Improved delivery can help accelerate research for emerging new therapies and help the understanding of disease pathways and mechanisms.  We will review multiple delivery solutions for CRISPR based technologies and also highlight protocol details and data outcomes for an iPSC specific CRISPR experiment.

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