Home Department: Human Biology and Political Science
Mentor: Jeffrey Goldberg (Ophthalmology)

“Optimization of Adeno-Associated Virus to Porcine Retinal Ganglion Cells and Serum Neutralizing Antibody Response ”

Traumatic optic nerve injury may lead to loss of vision with the death of retinal ganglion cells (RGCs), the neurons that transmit visual information from the eyes to the brain. RGCs are highly sensitive to injury, and like other neurons in the central nervous system, they cannot regenerate. Thus, injury may result in permanent blindness. While advancements in neuroprotection and regeneration have been made in rodent models, limited data in large animal models remains a challenge for developing human therapies. Ciliary neurotrophic factor (CNTF) and cyclic AMP (cAMP) promote RGC survival and regeneration in rodent models, and Ashley plans to research their role in a porcine model. This will include inducing a porcine model of optic nerve injury, administering CNTF and cAMP intravitreally, quantifying RGCs, and comparing these values to the vehicle control. This study will provide insight into the extent to which rodent treatments are translatable to larger species and whether CNTF and cAMP may be a viable therapeutic candidate in humans.