When the Human Genome Project published its blueprint of the human genetic code in 2001, life science researchers soon realized that unlocking the power of this research would next require detailed knowledge of the genetic variations that define individuals and how these predicted the onset or progression of disease as well as how patients would respond to novel targeted therapies.
The challenge of interpreting this wealth of information is being addressed by translational genomics researchers, who are now greatly aided by the ability to alter ‘at will’ the endogenous genome of human cells in culture using rAAV and nuclease-mediated genome-editing. The ability to routinely and flexibly engineer differentiated human cells, other mammalian cell types and human embryonic and induced pluripotent cells through genome-editing is having a powerful impact across a wide range of fields. This includes understanding the genetic basis of disease, allowing enhanced yields of biopharmaceuticals and driving the development and prescription of novel medicines targeted to specific patient populations.
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