Start It Themselves | GenomeWeb

Start It Themselves

A six-year-old girl has become the first person to receive gene therapy for Sanfilippo syndrome, Today reports. Patients with Sanfilippo lack a certain enzyme and cannot break down long-chain sugars, leading to progressive worsening of mental status — Today says the condition is sometimes called 'childhood Alzheimer's disease.' It's a rare recessive disorder, affecting one in 70,000 births.

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