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

1 点作者 corememory将近 8 年前

1 comment

corememory将近 8 年前
From the article:<p>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.