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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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Editorial Rating

8

Qualities

  • 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.

Take-Aways

  • Animal trials suggest that gene therapy is a promising treatment for the genetic causes of hearing loss.
  • People and animals with Usher syndrome have a genetic mutation that causes malformed hairs to grow in the inner ear.
  • In gene therapy, scientists use viruses as vehicles to transport healthy genes into the DNA of living patients.

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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