Key translational and drug development challenges, including insufficient light sensitivity, interspecies differences, immune responses to viral vectors and microbial proteins, and the lack of standardized clinical endpoints for patients with profound visual impairment are discussed.
Retinal degenerative diseases, including age‐related macular degeneration, retinitis pigmentosa, diabetic retinopathy, and inherited retinal dystrophies, are among the leading causes of irreversible vision loss and blindness worldwide. Current treatments primarily slow disease progression and are unable to restore lost...
This review critically compares the two approaches in spatial resolution, neural adaptation, and biosafety, while explicitly distinguishing clinical evidence from preclinical and proof-of-concept studies.
Na Long, Meng Si, Yingxian Jiang et al.· Frontiers in Medical Technol...· 0 citations
Glaucoma is a chronic neurodegenerative disease characterized by the deterioration of the optic nerve and the progressive loss of retinal ganglion cells (RGCs), making it a leading cause of irreversible blindness worldwide. This neurodegenerative process is characterized by the apoptotic death of neurons whose axons co...
Ya-Qin Zhang, Xiao-Yu Dong, Cai-Yan Zheng et al.· Current Neuropharmacology· 0 citations
Induced pluripotent stem cell (iPSC)-derived retinal organoids (ROs) have emerged as a platform for photoreceptor replacement therapy, supported by two complementary approaches: cone precursor cell transplantation and RO transplantation. Both have demonstrated proof of concept for functional synaptic integration with t...
This review discusses this emerging field and its transformative potential for translational neuropharmacology, focusing on therapies for neurodevelopmental and neurodegenerative disorders.
It is highlighted that oxidative stress contributes to inflammation, metabolic dysfunction, and cellular damage during disease progression and may be exacerbated by interventions associated with gene therapy—including vector-induced immune activation, transgene-related cellular stress, and delivery-associated tissue in...
