Full text is available at the source.
Lipid nanoparticle-mediated CRISPR/Cas9 delivery enables efficient trabecular meshwork gene editing in mice
Using lipid nanoparticles to deliver CRISPR for efficient gene editing in the eye's fluid drainage system in mice
AI simplified
Abstract
SM102-based lipid nanoparticles achieved the highest transfection efficiency for gene delivery to ocular tissues, particularly in the trabecular meshwork.
- Transfection efficiency of SM102-GFP was superior among three tested formulations in cultured ocular cells.
- After intravitreal injection in mice, GFP expression peaked in the trabecular meshwork one week later and could be re-induced with a second dose.
- SM102-GFP demonstrated better specificity for the trabecular meshwork and reduced retinal inflammation compared to adeno-associated viral and adenoviral vectors.
- Co-delivery of CRISPR components via SM102-based lipid nanoparticles enabled knockout of Matrix Gla Protein, influencing intraocular pressure and anterior chamber dynamics.
- Mgp knockout resulted in elevated intraocular pressure and signs of retinal stress, consistent with features of primary open-angle glaucoma.
AI simplified