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Gene Therapy Tackles a Common Birth Defect: Deafness
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Gene Therapy Tackles a Common Birth Defect: Deafness

After false starts, researchers are making progress toward treating deafness with gene therapy

Dina Fine Maron
Scientific American, 2018
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8

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  • Innovative
  • Scientific
  • Eye Opening

Recommendation

In 1999, an 18-year-old named Jesse Gelsinger tried an experimental therapy to treat his liver disorder. Using a virus as a vehicle, doctors at the University of Pennsylvania transported healthy genes into his DNA in an effort to rewrite faulty genetic programming. Gelsinger’s immune system responded by launching a violent offensive against his own organs. He died soon after. This wasn’t an auspicious beginning for gene therapy, but after 20 years of fine-tuning, scientists are now seeing success. Dina Fine Maron describes the hopeful results of animal trials in this Scientific American article which will fascinate anyone concerned with the future of gene therapy.

Summary

Animal trials suggest that gene therapy is a promising treatment for the genetic causes of hearing loss.

Most mice are enthusiastic swimmers. The first time they’re tossed into a body of water, they know just what to do. But certain mice in a lab at Boston Children’s Hospital are at a loss when they encounter water. They “frantically paddle in circles for a few seconds, struggling to determine which side is up.” They also recoil to the corner of a cage rather than sniffing around, and fail to respond to loud noises.

These mice have been bred to have mutations associated with hearing loss. But just months after a new, experimental gene therapy, these...

About the Author

Dina Fine Maron, formerly an associate editor at Scientific American, is now a wildlife trade investigative reporter at National Geographic.

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