Gene-Silencing: ‘New Class’ of Medicine Reverses Disease Porphyria

April 15, 2019

(BBC) – Doctors have used a new type of medicine called “gene silencing” to reverse a disease that leaves people in crippling pain. The condition, acute intermittent porphyria, also causes paralysis and is fatal in some cases. The novel approach fine-tunes the genetic instructions locked in our DNA. Doctors say they are “genuinely surprised” how successful it is and that the same approach could be used in previously untreatable diseases.

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