Gene therapy for neuroprotection and axon regeneration in CNS injuries
Innovative gene therapy using AAV2 vectors to target Dynlt1a and Lars2 offers potential neuroprotection and axon regeneration for CNS neurons, specifically retinal ganglion cells affected by optic neuropathies. This approach aims to restore visual function after optic nerve injuries.
Overview
This cutting-edge gene therapy aims to address the lack of spontaneous axon regeneration in CNS neurons, specifically retinal ganglion cells (RGCs), following optic neuropathies such as optic nerve trauma, ischemia, and glaucoma. The proposed solution involves the use of AAV2 vectors to deliver genes targeting Dynlt1a and Lars2, which are associated with mitochondrial functions. These targets have shown promise in promoting neuroprotection and axon regeneration without the tumorigenic risks associated with other genetic targets, such as Pten. Successful regeneration could lead to restored visual functions, providing a significant breakthrough in treating optic nerve injuries.
Technical specifications
- Gene Targets: Dynlt1a and Lars2, chosen for their roles in mitochondrial-related neuroprotection and regeneration
- Delivery Method: Intravitreally injected AAV2 vectors, ensuring localized and efficient gene delivery to RGCs
- Validation Procedures: RGC survival and axon regeneration will be assessed through standard methods, including axon tracing and immunostaining with confocal microscopy, over a 3-month period post-injury
- Preclinical Model: Mouse CNS injury model with optic nerve crush (ONC) to evaluate the efficacy of the gene therapy
Technology readiness level
Currently at TRL 3, this technology is in the experimental proof-of-concept stage. Validation is being conducted using established methods in preclinical models, with future plans to assess functional restoration of visual capabilities.
About University of Connecticut
UConn is a comprehensive public research university and the state’s flagship, with its main campus in Storrs and an academic medical center in Farmington. Industry engages through UConn Tech Park—anchored by the Innovation Partnership Building—with shared labs, pilot‑scale testbeds, and advanced prototyping for collaborators. Integration with UConn Health enables clinical collaborations and translation, while Hartford and Stamford campuses connect companies to talent along the New York–Boston corridor; a statewide extension network supports technology adoption. Research is supported by competitive federal funding from agencies such as NIH, NSF, DOE, USDA, and DOD. A dedicated technology commercialization office and the Technology Incubation Program support IP, licensing, and startup incubation across multiple sites.
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