
Huntington’s Disease: Gene Editing Shows Promise in Mouse Studies - corememory
https://directorsblog.nih.gov/2017/06/27/huntingtons-disease-gene-editing-shows-promise-in-mouse-studies/
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corememory
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.

