[cartoon id= a14926"] Latham's survival instinct has no clear genetic root, nor does his equal and opposite tendency to throw himself into harm's way.
By being able to insert DNA into existing cells, scientists can "attack disease at its genetic root and turn off receptors that regulate how a cell functions, stopping a disease pathway in its tracks," explains Mirkin.
She and her parents, Kelly and Craig Taws – who have travelled from Emsworth in Hampshire to south London to see specialists – have been seeing doctors since Evie was seven months old, because of a range of symptoms that clinicians now believe has a genetic root.
But it would take the technology decades to sequence enough genomes necessary to begin isolating the genetic root of many diseases.
Van Goor's chutzpah led him on a decade-long quest involving dozens of scientists that resulted in the first drug to treat the disease at its genetic root.
Though far from producing a "smoking gun," the findings provide substantial support for a genetic root to the disorder and greatly narrow the search for genes that may cause the disease, the authors said.
