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Extracellular Vesicles in Ophthalmology: From Natural Nanocarriers to Engineered Therapeutics
Christopher Flores1,2, Fabiana Mastantuono1,3, Lu Huang1
1Department of Ophthalmology, Schepens Eye Research Institute of Mass Eye and Ear, Harvard Medical School, Boston, MA 02114, USA.
Abstract:
Extracellular vesicles (EVs) are increasingly recognized as programmable bioactive carriers in non-viral gene delivery and adaptable bioengineering platforms. Beyond their roles as natural nanocarriers in intercellular communication, EVs can promote ocular surface repair and retinal neuroprotection with potential for low immunogenicity and high biocompatibility. Bioengineering now enables cargo encapsulation, surface targeting, and integration of EVs with biomaterial platforms to enhance tissue penetration, retention, and precision delivery. The emergence of induced pluripotent stem cell-derived EVs (iMSC-EVs) offers improved batch uniformity and potential for personalized therapy. However, progress hinges on resolving knowledge gaps in ocular EV biology, standardizing isolation and storage, scaling reproducible manufacturing, and executing focused clinical trials. We synthesize the current developments and outline how EVs are moving from biological mediators to engineered therapeutics to accelerate the translation of EV diagnostics and therapeutics for eye diseases.
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