Harvard Medical School
For 30 years, researchers have known that Huntington's disease is caused by an inherited mutation in the Huntingtin (HTT) gene in which a three-letter DNA sequence, C-A-G, is repeated at least 40 times. But they didn't know how the mutation behind this fatal neurodegenerative disorder causes brain cell death, why only some brain cells die and not others, and why most patients develop symptoms only in middle age, after decades of apparent good health.
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