Therapeutic Applications in Next-Generation Biopharmaceuticals: Enhanced LYTAC Activity and Genome Editing Efficiency (IMAGE)

Kumamoto University

Therapeutic Applications in Next-Generation Biopharmaceuticals: Enhanced LYTAC Activity and Genome Editing Efficiency

Caption

Evaluation of the polyrotaxane delivery technology in advanced therapeutic models. (a) Administration of the polyrotaxane-applied LYTAC in mice demonstrated a significantly higher therapeutic effect compared to conventional LYTAC equipped with traditional triGalNAc. (b) Conjugation of monoGalNAc-modified polyrotaxane to Cas9 RNP-loaded nanoparticles successfully improved genome editing efficiency of the target transthyretin (ATTR amyloidosis-causing) gene in the liver.

Credit

Taharabaru et al.

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CC BY-NC-ND

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