non-invasive stem cell therapy in a rat model for retinal degeneration and vascular pathology非侵入性的干细胞疗法在老鼠模型中对视网膜变性和血管病变.pdfVIP

Non-Invasive Stem Cell Therapy in a Rat Model for Retinal Degeneration and Vascular Pathology 1 1 1 1 1 2 Shaomei Wang *, Bin Lu , Sergei Girman , Jie Duan , Trevor McFarland , Qing-shuo Zhang , Markus 2 1 1 1 Grompe , Grazyna Adamus , Binoy Appukuttan , Raymond Lund 1 Casey Eye Institute, Oregon Health Science University, Portland, Oregon, United States of America, 2 Oregon Stem Cell Center, Oregon Health Science University, Portland, Oregon, United States of America Abstract Background: Retinitis pigmentosa (RP) is characterized by progressive night blindness, visual field loss, altered vascular permeability and loss of central vision. Currently there is no effective treatment available except gene replacement therapy has shown promise in a few patients with specific gene defects. There is an urgent need to develop therapies that offer generic neuro-and vascular-protective effects with non-invasive intervention. Here we explored the potential of systemic administration of pluripotent bone marrow-derived mesenchymal stem cells (MSCs) to rescue vision and associated vascular pathology in the Royal College Surgeons (RCS) rat, a well-established animal model for RP. Methodology/Principal Findings: Animals received syngeneic MSCs (1 6106 cells) by tail vein at an age before major photoreceptor loss. Principal results: both rod and cone photoreceptors were preserved (5–6 cells thick) at the time when control animal has a single layer of photoreceptors remained; Visual function was significantly preserved compared with controls as determined by visual acuity and luminance threshold recording from the s