🧬 New gene editing technique may have cured one-year-old's deadly disease

• A child with a deadly metabolic disease has been successfully treated with a new type of gene editing called ARCUS.
• The treatment allowed the child to stop their special diet after three months.
• The technique succeeded in getting up to 30 percent of liver cells to start producing the missing enzyme in animal trials.

First patient responds positively to treatment

A one-year-old boy with the deadly disease OTC deficiency has been treated with a new type of gene editing. After treatment, the boy, who had previously suffered from two seizures due to elevated ammonia levels, was able to stop his special diet after three months, reports Science.

OTC deficiency is a genetic disease that prevents the body from breaking down ammonia in the blood. This leads to seizures, brain damage and can be fatal. Currently, only liver transplantation exists as a cure for infants with early OTC deficiency.

The treatment consisted of two types of AAV viruses. One virus carried the DNA for ARCUS and the other carried the gene for OTC. The gene was placed near the PCSK9 gene in the liver, a location chosen to minimize the risk of interference with the rest of the DNA.

The company iECURE, which developed the treatment, will present complete data in March. They are now planning to treat more patients with the same dose after the positive results from the first patient.

New technology enables permanent change

The new technique, ARCUS, is a DNA-cutting enzyme that comes from algae. Unlike CRISPR, ARCUS doesn't need a guide RNA sequence to find the right location in DNA. ARCUS is also smaller than CRISPR which makes it easier to deliver to cells.

In studies on monkeys, including infants, the technique achieved good results. Up to 30 percent of liver cells began producing the OTC enzyme, which was sufficient to break down ammonia in the blood. The effect persisted after one year in infants.

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