You may have heard in the last two weeks that scientists successfully restored sight to three blind dogs suffering from a rare genetic disease simply by injecting a healthy gene. Studies are now under way to see if that treatment could be effective in humans suffering from a version of the same disease, which causes total blindness in infancy. This is the merely the tip of the iceberg for gene therapy, which will present some of the most exciting opportunities of the 21 st century for doctors, patients and investors.
Gene therapy is an amazing technology.
The goal is to get us to the point where each cell acts like a little factory producing the needed therapy so it doesn't have to be injected. The recent sequencing of the human genome and increased understanding of the role certain genes play in disease kick the door wide open for gene therapy to advance faster and smarter than ever. There are now whole biotech companies founded on the concept of identifying genes and relating them to their corresponding diseases. As always, the biggest profits will come from companies with the best science. Viruses are most often used as vectors, but they're often stumbling blocks as well.