CRISPR Therapeutic Applications

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A protein structure of ABE8e is shown, superimposed with cartoon representations of sgRNA and target DNA. Two TadA domains are highlighted. The catalytic domain contacts multiple bases of the non-target DNA strand, while the docking domain primarily contacts the other protein domains.
A smiling Blugene holds DNA
Schematic of a human body with cartoons indicating systemic therapy (via IV); patient-derived editing ex vivo (via cell culture); and allogenic therapy (via cells derived from another person).
CRISPR embryo repair
Decorative image showing a bacteriophage and CRISPR versus angry bacteria.
exon skipping strategies for Duchenne muscular dystrophy

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