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

Abstract

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 targeting other hemoglobinopathies and immune deficiencies are currently under evaluation in clinical trials [ 2 ].

Topics & Concepts