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Beam Therapeutics has provided a much-awaited update on its therapy that uses a form of CRISPR called base editing to precisely fix a single genetic typo responsible for a rare genetic disease.
On Wednesday, Beam said it has now treated 29 patients with alpha-1 antitrypsin deficiency (AATD), a condition caused by a broken enzyme, AAT, that is normally made in the liver and is vital for protecting the lungs.
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