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Next-generation genome editing tools surpass CRISPR milestone

Modifying a patientโ€™s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as ฮฒ-thalassemia and severe sickle cell disease (SCD). But science does not stop.
Jennifer Doudna Emmanuelle Charpentier Vertex Pharmaceuticals Inc Sickle Cell Disease Spinal Muscular Atrophy Base Editing

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