Gene Editing

CRISPR Genome Editing and the Future of Down Syndrome Treatment

The ethics of using safe gene therapies to improve the health and cognition of Down syndrome children and adults.

Ronald Bailey | 4.30.2026 11:30 AM

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Deaf children can now hear thanks to a new treatment that repairs a defective gene. Researchers associated with the biotech company Regeneron Pharmaceuticals unveiled the successful gene therapy last week. One patient, who was treated at 18 months old, Travis Smith, can now hear. Overall, 80 percent of participants (aged 10 months to 16), saw significant improvement in hearing, and 42 percent achieved normal hearing, including the ability to hear whispers. The Food and Drug Administration (FDA) has already approved the treatment.

Medical research on ameliorating physical and intellectual maladies continues apace. For example, researchers at Harvard Medical School reported earlier this month that they have made progress in silencing the extra chromosome that causes Down syndrome using the genome editing technique CRISPR.

Down syndrome is a genetic condition in which a person has an extra copy of chromosome 21. While its severity varies, people with the condition experience lifelong intellectual disability and developmental delays, along with heart and digestive system problems and a significantly higher risk of Alzheimer's disease.

CRISPR techniques have recently been successfully applied to treating other genetic conditions, including sickle cell anemia and a rare inborn error of metabolism of the........

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