Evaluating CRISPR Base Editing Workflows for Durable In Vivo Programming
- Optimizing base editor and payload design for efficient liver‑targeted delivery enabling durable, single‑dose functional gene inactivation in vivo
- Assessing off‑target, bystander and edit‑outcome profiles using discovery‑stage analytics strengthening confidence in precision and long‑term safety for clinical advancement
- Aligning potency and comparability strategies with modality‑specific editing dynamics streamlining CMC readiness and accelerating progress toward lasting therapeutic benefit