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Is it possible to overstate the potential of a new technology that efficiently and cheaply permits deliberate, specific, and multiple genomic modifications to almost anything biological? What if that technology was also capable of altering untold future generations of nearly any given species—including the one responsible for creating it? And what if it could be used, for better or worse, to rapidly exterminate entire species?
CRISPR-Cas9, of course, is only one among many prokaryotic CRISPR systems that could, at some point, prove useful for any number of human purposes. Use of Cas9 variations, however, has already resulted in successes far too numerous to review liberally here. Even so, two recent applications in particular reveal the extraordinary, yet strikingly simple, means by which researchers have achieved previously unattainable outcomes.
In the first, three different teams confronted Duchenne muscular dystrophy (DMD), a terrifying disease that affects about one in every 3500 boys in the U.S. alone. DMD typically stems from defects in a gene containing seventy-nine protein-coding exons. If even a single exon suffers a debilitating mutation, the gene can be rendered incapable of producing dystrophin, a vital protein that protects muscle fibers. Absent sufficient dystrophin, both skeletal and heart muscle will deteriorate. Patients usually end up confined to wheelchairs and dead before the age of thirty.
Read full, original post: CRISPR-Cas9: Not Just Another Scientific Revolution