siRNA drugs offer cure for single-gene liver diseases

david_stillwell · x · 2026-08-26

siRNA (small interfering RNA) drugs are transforming medicine by switching off disease-causing proteins instead of blocking them, bypassing the 'undruggable' problem of traditional chemicals. Diseases caused by single-gene action in the liver are now effectively solvable. Conditions like Huntington's, haemophilia, and potentially Alzheimer's may soon be treatable with a few yearly doses.

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