A rare disease cured by genetic scissors

The marriage of the Crispr-Cas9 technique with technologies similar to RNA vaccines gives very promising results in the face of a very rare but fatal liver disease, hereditary transthyretin amyloidosis.

For the first time, a genetic disease could be corrected directly in humans thanks to the use of the Crispr-Cas9 molecular scissors. This result was obtained barely nine years after the development of this genetic manipulation technique by the French Emmanuelle Charpentier and the American Jennifer Doudna (who received the Nobel Prize in chemistry in 2020 for this work) and six months after. a first successful application in ex vivo gene therapy in patients with sickle cell anemia.

It is a very rare but fatal genetic disease, hereditary transthyretin amyloidosis, which has been successfully treated with Crispr-Cas9, American researchers announced in the medical journal. The New England Journal of Medicine . This disease is caused by the accumulation in the body, including the heart and nerves, of a blood protein, transthyretin, produced by the liver and which has become toxic following a mutation in its gene. Death occurs

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