2. Academic Validation
  3. Promoting retinal ganglion cell regeneration with targeted liposome-based delivery of MHY1485 for optic nerve repair

Promoting retinal ganglion cell regeneration with targeted liposome-based delivery of MHY1485 for optic nerve repair

  • J Control Release. 2025 Jul 10:383:113778. doi: 10.1016/j.jconrel.2025.113778.
Bingqing Luo 1,  Tingting Cheng 2,  Ying Xiang 1,  Shengkai Sun 3,  Yuanshan Liu 3,  Jincong Yan 1,  Yajie Zhang 3,  Yi Cao 1,  Xingzhu Liu 4,  Renjun Pei 5
Affiliations

Affiliations

  • 1 School of Nano-Tech and Nano-Bionics, University of Science and Technology of China, Hefei 230026, China; CAS Key Laboratory for Nano-Bio Interface, Suzhou Institute of Nano-Tech and Nano-Bionics, Chinese Academy of Sciences, Suzhou 215123, China.
  • 2 CAS Key Laboratory for Nano-Bio Interface, Suzhou Institute of Nano-Tech and Nano-Bionics, Chinese Academy of Sciences, Suzhou 215123, China; Department of Chemistry, College of Sciences, Shanghai University, Shanghai 200444, China.
  • 3 CAS Key Laboratory for Nano-Bio Interface, Suzhou Institute of Nano-Tech and Nano-Bionics, Chinese Academy of Sciences, Suzhou 215123, China.
  • 4 CAS Key Laboratory for Nano-Bio Interface, Suzhou Institute of Nano-Tech and Nano-Bionics, Chinese Academy of Sciences, Suzhou 215123, China. Electronic address: xzliu2020@sinano.ac.cn.
  • 5 School of Nano-Tech and Nano-Bionics, University of Science and Technology of China, Hefei 230026, China; CAS Key Laboratory for Nano-Bio Interface, Suzhou Institute of Nano-Tech and Nano-Bionics, Chinese Academy of Sciences, Suzhou 215123, China. Electronic address: rjpei2011@sinano.ac.cn.
PMID: 40288495 DOI: 10.1016/j.jconrel.2025.113778
Abstract

Optic neuropathy, characterized by the irreversible degeneration of RGCs, leads to vision loss for which treatment options remained limited. Nanoparticle-based therapeutic strategies have been developed to stimulate RGCs regeneration; however, insufficient nanoparticles delivery to RGCs and a lack of robust stimulation of RGCs intrinsic regenerative capacity, have limited their application. Here, we demonstrate that a RGC-targeting TET1 conjugated to Liposome delivery system (Lipo-T) enhances therapeutic agent delivery to RGCs while minimizing off-target effect for activating glial cells. Targeted delivery of a mTOR-specific activator MHY1485 to RGCs via Lipo-T (MHY@Lipo-T) significantly enhances in vivo RGCs survival, neurite outgrowth, and axonal regeneration without retinal inflammation from unwanted glial activation. By conferring sustained MHY1485 to degenerated retina through Lipo-T injection, and with its high target ability potential, the first use of MHY@Lipo-T expands new therapeutic option for optic nerve injury.

Keywords

Liposome; MHY1485; Regeneration; Retinal ganglion cells; Targeted delivery.

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