Imaging guide stem cells and gene therapy for retinal diseases (Conference Presentation)

Retinal diseases such as age-related macular degeneration (AMD), retinal pigmentary disorders, and diabetic retinopathy are leading causes of vision loss worldwide, with current treatments offering limited efficacy in improving vision. In this study, we present an advanced multimodal imaging system that integrates photoacoustic microscopy (PAM), optical coherence tomography (OCT), and fluorescent imaging into a unique platform for tracking stem cell viability, migration, and replacement of damaged retinal tissue. Additionally, this system was employed to monitor adeno-associated virus (AAV) transfection for gene therapy. Experiments were conducted on 21 rabbits with clinically relevant models such as laser-indced retinal pigment epithelium (RPE) damage. Results demonstrated that the transplanted stem cells successfully grafted onto the rabbit retina and progressively replaced damaged RPE over six months. Furthermore, the efficiency of cell transfection was detectable via OCT and fluorescent imaging approximately two weeks following subretinal injection of AAV serotypes 2, 5, and 8. These findings pave the way for new treatment avenues for various retinal diseases.