Article
Lipid-nanoparticle-mediated base editing of the trabecular meshwork rescues glaucoma in vivo.
JCI insight - 9 Feb 2026
Kaipa Balasankara Reddy, Li Linya, Gopalakrishnan Prakadeeswari, Du Samuel, Felgner Jiin, Palczewski Krzysztof, Felgner Philip, Zode Gulab S
Abstract excerpt
Mutations in MYOC, the most common genetic cause of glaucoma, cause misfolded myocilin to accumulate in the endoplasmic reticulum (ER), leading to trabecular meshwork (TM) dysfunction, elevated intraocular pressure, and progressive vision loss. While gene editing offers curative potential, current delivery methods rely on viral vectors, which are limited by inflammation, off-target effects, and poor...
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