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Huntington’s Disease: Gene Editing Shows Promise in Mouse Studies

directorsblog.nih.gov

1 points by corememory 8 years ago · 1 comment

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corememoryOP 8 years ago

From the article:

Huntington’s disease occurs when a person inherits from one parent a mutant copy of the huntingtin (HTT) gene that contains extra repetitions, or a “stutter,” of three letters (CAG) in DNA’s four-letter code. This stutter leads to production of a misfolded protein that is toxic to the brain’s neurons, triggering a degenerative process that, over time, leads to mood swings, slurred speech, uncontrolled movements, and, eventually, death.

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