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What is Genome Editing?

Genome editing is a technique that attempts to edit a specific gene in the DNA. Sangamo’s approach to genome editing involves the use of zinc finger nucleases (ZFNs), which are naturally occurring proteins that can bind to and edit very precise locations of DNA in the human body.

 

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In the Thales Research Study, participants' blood stem cells are collected from the bloodstream and are then shipped to a lab where the DNA of those cells will be precisely edited. This works as follows: uniquely engineered ZFNs make a specific cut and modify the DNA at a targeted location in the BCL11A gene without significantly disrupting other areas of DNA. If successful, the edit in the BCL11A gene will allow red blood cells to produce more fetal hemoglobin and reduce or remove the need for regular blood transfusions.  


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