Neurotrophic factor genome engineering could have many potential applications not only in the deeper understanding of
neurodegenerative disorders but also in improved
therapeutics. The fields of nanomedicine, regenerative medicine, and gene/cell-based
therapy have been revolutionized by the development of safer and efficient non-viral technologies for gene delivery and genome editing with modern techniques for insertion of the
neurotrophic factors into clinically relevant cells for a more sustained
pharmaceutical effect. It has been suggested that the long-term expression of
neurotrophic factors is the ultimate approach to prevent and/or treat
neurodegenerative disorders such as
glaucoma in patients who do not respond to available treatments or are at the progressive stage of the disease. Recent preclinical research suggests that novel neuroprotective gene and cell
therapeutics could be promising approaches for both non-invasive neuroprotection and regenerative functions in the eye. Several progenitor and
retinal cell types have been investigated as potential candidates for
glaucoma neurotrophin therapy either as targets for gene therapy, options for cell replacement
therapy, or as vehicles for gene delivery. Therefore, in parallel with deeper understanding of the specific protective effects of different
neurotrophic factors and the potential therapeutic cell candidates for
glaucoma neuroprotection, the development of non-invasive and highly specific gene delivery methods with safe and effective technologies to modify cell candidates for life-long neuroprotection in the eye is essential before investing in this field.