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Daring to Hope

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Daring to Hope

Patients thrill to reports of a promising antisense drug against Huntington disease, but no one is sure yet whether it works

Science,

5 mins. de lectura
3 ideas fundamentales
Audio y Texto

¿De qué se trata?

A new drug for Huntington disease seems to be safe, but its efficacy is yet to be determined.

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Editorial Rating

8

Qualities

  • Scientific
  • Applicable
  • Inspiring

Recommendation

Michelle Dardengo watched her father’s body, personality and life deteriorate after he was diagnosed with Huntington disease at age 52. It changed him from a music-loving volunteer firefighter into a twitching, suicidal alcoholic. When she turned 52, she received the same diagnosis. But in the intervening decades scientists had developed a drug: An antisense DNA molecule that disables the mutant gene responsible for the disease. Dardengo participated in the first clinical trial, which demonstrated that the drug is safe. But it remains to be seen if it will be effective. The article will compel anyone who likes the ways advances in science can lead to stories of hope.

Summary

Huntington disease precipitates a slow decline in people’s physical and mental faculties.

Huntington disease is brutal. It strikes people in midlife and slowly robs them of their ability to control their bodies and minds. Huntington disease is inherited in an autosomal dominant manner, so the chances of passing it on to the offspring are 50 percent.

A mutation in the so-called huntingtin gene (which encodes the huntingtin protein) is thought to cause Huntington disease. Healthy people’s version of the gene contains fewer than 35 copies of the genetic sequence CAG – cytosine, adenine, guanine – which encodes the amino acid glutamine. Huntington patients’ mutant versions contain 36 or more of these CAG repeats, yielding longer glutamine chains in the...

About the Authors

Meredith Wadman is a reporter at Science magazine. She has also written on biotech and on biomedical policy issues for Time, The New York Times, The Washington Post and The Wall Street Journal. Wadman is the author of The Vaccine Race: Science, Politics and the Human Costs of Defeating Disease.

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