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This story has been mentioned in last night’s Overnight News Digest, but not elsewhere on this site.
Since the discovery of the molecular machinery behind heredity, it has been a dream of physicians and medical geneticists to be able to cure genetic diseases by modifying the defective gene. That day has now arrived. An infant, now 9 months old, was treated for a rare, life-threatening condition using gene therapy mediated by CRISPR, and he appears to be doing fine. The infant’s condition is called CPS1.
CPS1 deficiency is characterized by an inability to fully break down byproducts from protein metabolism in the liver, causing ammonia to build up to toxic levels in the body. It can cause severe damage to the brain and liver. Treatment includes a low protein diet until the child is old enough for a liver transplant. However, in this waiting period there is a risk of rapid organ failure due to stressors such as infection, trauma, or dehydration. High levels of ammonia can cause coma, brain swelling, and may be fatal or cause permanent brain damage.
The infant was treated at Children’s Hospital of Philadelphia, using a treatment developed at the Perelman School of Medicine at the University of Pennsylvania. Therapy was based on CRISPR, genetic machinery that enables gene editing. By administering therapy to the liver, the team was able to edit the infant’s genetic defect, which made his liver capable of breaking down proteins without producing toxic levels of ammonia.
The child initially received a very low dose of the therapy at six months of age, then a higher dose later. The research team saw signs that the therapy was effective almost from the start. The six-month old began taking in more protein in the diet, and the care team could reduce the medicine needed to keep ammonia levels low in the body.
Significant also was the fat that, twice during treatment, he became ill, once with a cold, and again with an intestinal ailment. Such illness can be extremely dangerous for children with this condition, but rather than inducing a crisis, the infant just shrugged them off.
This infant will have to be observed over the long-term, as he grows, to see if there are any ill effects due either to any residual elements of his genetic condition, or to the treatment. However, when it comes to treating genetic diseases, a new day has dawned. Those suffering from the consequences of genetic diseases can hope that their conditions can be cured.
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