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Genome editing technology approved to target rare blood disorders

This is an anointing for the American Vertex laboratory. Following Britain last month, the United States on Friday gave the green light to its Casgevy treatment for sickle cell anemia, the world’s first to be developed using Crispr, a genome-editing technology. This method, known as “molecular scissors”, makes it possible to carry out targeted changes in the genetic material of a cell.

It awarded the Nobel Prize in Chemistry to Frenchwoman Emmanuelle Charpentier and American Jennifer Doudna in 2020. This important medical advance holds great promise for the development of other treatments »American President Joe Biden greeted on Friday. Just eleven years after these researchers published their first paper in the journal Sciencea cure has emergedsays Reshma Kewalramani, CEO, Vertex, a nephrologist by training. Search time has increased. »

Sickle cell anemia, the “least rare of the rare diseases,”…

Source: Le Figaro

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