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Updated: Dec 26, 2025

Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
[Recent advances in Huntington's disease]
Valter Niemelä1, Åsa Petersén2, Ghada Loutfi3
1Akademiska sjukhuset - Neurologiska kliniken Uppsala, Sweden Akademiska sjukhuset - Uppsala, Sweden.
Abstract:
Huntington's disease is an autosomal dominant neurodegenerative disease that leads to premature death. The disease is caused by a pathological CAG triplet expansion in the huntingtin gene. The disease is most common in Western populations, with onset in middle age and causing progressive motor, cognitive, and psychiatric symptoms. Currently, only symptomatic treatment is provided, but new molecular technologies may allow treatments reducing levels of mutated huntingtin.
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