With the first CRISPR–Cas9 gene therapy now approved, scientists are turning to newer editing technologies to produce safer, faster and better treatments for genetic diseases.
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Going from the laboratory to an approved therapy in 11 years is no mean feat. That is the story of the world’s first approved CRISPR–Cas9 therapy,in December 2023. Casgevy , from Vertex and CRISPR Therapeutics, aims to cure sickle-cell disease in a ‘one and done’ treatment. Sickle-cell disease causes debilitating pain and organ damage that can lead to life-threatening disabilities and early death.
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