SMRTR Science & EngineeringSep 24, 2025The Guardian

Huntington’s disease treated successfully for first time in UK gene therapy trial

SMRTR summary

A groundbreaking gene therapy trial has successfully treated Huntington's disease for the first time in the UK, slowing disease progression by 75% after three years. The single-shot treatment inactivates the toxic protein causing this inherited condition that leads to dementia, paralysis, and death. This breakthrough offers hope to thousands of UK patients and may encourage more at-risk individuals to seek genetic testing, with treatment potentially preventing symptoms if administered early enough.

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