We could also use this to actually change DNA sequences or correct them, and much of our monogenic disease particularly in the areas of hemophilias are involved with this. We could also and finally use this to insert large sequences of DNA into so-called safe harbor locations in a genome. We can do this in plants, we can do this in humans, and so it’s a very broad, very general technology platform that has very unique opportunities in the area of human health care.And so, when you think about the technology platform and you start to filter that through the kinds of therapeutic programs that can be developed, we can think about this in two large buckets, direct in vivo applications and modified cells or ex vivo applications. And while I won’t go through all of these today, this begins to give you a sense of how we think about the opportunity to leverage this very general, very robust, gene-specific, gene-modification, gene-regulation technology platform to create highly differentiated, and in many cases, therapeutics that are not just intended to treat diseases, but actually physically and permanently correct or change DNA sequences in a way that can engineer genetic cure. And that’s really the ultimate goal of the technology.
Sangamo BioSciences' CEO Presents At The 2012 Wedbush PacGrow Lifesciences Management Access Conference (Transcript)
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