Genetic engineering offers the promise of treating genetic disorders by correcting disease-causing DNA, but when the defective genes are located in cells deep inside the body, how do you actually deliver a cure?
Over the past year, the Food and Drug Administration has approved threelandmark therapies that work by genetically modifying a patient’s cells, but those therapies have all focused on cells that are easy to reach, like those in the blood. A new study in mice offers a glimpse at how researchers might use gene therapy on harder-to-reach cells—in this case, the motor neurons in the spinal cord responsible for amyotrophic lateral sclerosis, or Lou Gehrig’s disease.
ALS is a fatal neurodegenerative disease. Within three to five years of the first symptoms appearing, progressive weakening of the body’s muscles eventually leads to paralysis and death. There is no cure. The very best drugs on the market extend survival by only a few months.
Most cases of ALS disease are not inherited, but in a small percentage of cases, we know the exact gene responsible for causing its...