In a groundbreaking development, doctors in the United States have successfully treated a baby with a rare, life-threatening genetic disorder using a tailor-made gene-editing therapy.

The procedure, which was developed in just six months, uses technology that enables doctors to precisely target and correct a faulty gene in the child’s DNA.

The baby, known as KJ, was diagnosed with Carbamoyl Phosphate Synthetase 1 deficiency, a condition that prevents the body from processing ammonia, leading to potential brain damage or death. Doctors at the Children’s Hospital of Philadelphia and the University of Pennsylvania created a personalised base editor to fix the mutation in KJ’s liver cells.

After receiving three doses of the therapy, KJ showed significant improvements, including increased consumption of protein and reduced need for ammonia-reducing medications.

While the treatment isn’t a complete cure, it offers hope for patients with rare genetic disorders, demonstrating the potential of DNA-altering techniques in preventing organ damage and improving life expectancy.

Doctors are optimistic that KJ’s progress could pave the way for future gene-editing treatments for similar conditions, transforming how rare diseases are treated.

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