In vivo gene editing delivers the CRISPR machinery directly into the patient's body, editing target cells where they naturally reside instead of removing, editing, and re-infusing cells outside the body.
edit success ~ deliveryEff * precision, off-target risk ~ (1-precision)
- RNP complexes delivered — Cas9 protein pre-complexed with guide RNA, packaged in a lipid nanoparticle or viral vector for delivery.
- Genomic target sites — the disease-relevant loci (e.g. PCSK9, TTR) targeted directly inside the patient's own tissue.
- Delivery efficiency — fraction of target cells actually reached and successfully transfected by the editing complex.
- Editing precision — how selectively the complex cuts only its intended site versus off-target genomic locations.
NTLA-2001, the first in vivo CRISPR therapy to show durable effect in humans, works by exactly this route: lipid-nanoparticle-delivered Cas9/guide RNA edits liver cells directly inside the patient, no cell removal required.