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A first for CRISPR: powerful prime editing alters DNA directly in the human body
Nature • • health
Key Points:
- Prime Medicine has conducted the first known in-body gene-editing treatment using prime editing to directly rewrite DNA in a patient's liver cells, targeting Wilson’s disease by correcting mutations that cause copper buildup.
- Prime editing offers greater precision than traditional CRISPR–Cas9 methods and can insert, delete, or rewrite DNA segments, making it suitable for correcting mutations that base editing cannot address.
- The in vivo approach of editing DNA directly inside the body could reduce costs and speed up treatment compared to the standard method of editing cells outside the body and reinjecting them.
- Wilson’s disease, caused by mutations in the ATP7B gene, currently requires complex lifelong treatments, and gene editing holds promise as a one-time, lasting solution.
- The FDA has approved only one gene-editing therapy so far (Casgevy), which uses CRISPR–Cas9 to disable genes; prime editing represents a more versatile advancement in gene therapy technology.
