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New gene-editing approach shows promise in treating Huntington's disease in mice

Huntington's disease is caused by repetitive "stutters" in DNA—specifically repeated CAG sequences—that expand abnormally over time, leading to toxic protein accumulation that contributes to the progression of the disease. There is currently no cure for this rare and fatal genetic disease.

Published by
Medical Xpress
Evidence type
Animal

Huntington's disease is caused by repetitive "stutters" in DNA—specifically repeated CAG sequences—that expand abnormally over time, leading to toxic protein accumulation that contributes to the progression of the disease. There is currently no cure for this rare and fatal genetic disease.

This item was selected automatically by our daily literature and news monitor because it matches the topics we follow. Read the full story at Medical Xpress.

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New gene-editing approach shows promise in treating Huntington's disease in mice | Brilliant Blue Biosciences