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Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute

1 Nov 2023

Abstract excerpt

For individuals living with debilitating hereditary diseases, therapies that can modify a person’s genome (genomic therapies) hold the promise to shift treatment outlooks from a lifetime of chronic disease management to a cure [ 1 ]. We now have groundbreaking therapies for multiple disorders, including genetic eye diseases, spinal muscular atrophy, and beta-thalassemia (Table 1 ). Additional genomic therapies...

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PMID: 37935852
DOI: 10.1038/s41434-023-00392-3

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