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New-generation Guide RNA (LgRNA) Platform
- Efficacy: Dual guide RNAs < sgRNA < LgRNA
- LgRNA is highly pure (up to 99% by LC/MS) and free of detectable truncated spacer sequences
- High purity results in very high specificity
- Chemical modifications of LgRNA limit immunogenicity
- LgRNA conjugates enable nucleus uptake and cell-selective delivery
Non-immunogenic STAR Editor Platform
- Precise editing by a DNA dependent polymerse
- nCas9/templated DNA repair resulting in minimal DSBs
- RNP conjugates for non-immunogenic targeted delivery
- Ability to insert stop codon anywhere to ensure minimal truncated protein byproducts for anti-cancer and antiviral therapy
- Ability to perform single base corrections, and insert/delete short or long DNAs
- Capability of multiplexing and multiple-dosing
