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Development of non-invasive cell-based therapy for retinal degeneration and assoc

开发针对视网膜变性及其相关的非侵入性细胞疗法

基本信息

批准号：
8597428
负责人：
Shaomei Wang
金额：
$ 40.92万
依托单位：
CEDARS-SINAI MEDICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
起止时间：
2011-01-01 至 2015-12-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8597428
关键词：
Animal Model Area Autologous Back Blindness Blood Vessels Bone Marrow CXCR4 gene Cell Adhesion Molecules Cell Survival Cell Therapy Cells Cessation of life Clinic Clinical Clinical Trials Development Disease Dose Effectiveness Encapsulated Ethics Eye Genetic Growth Factor Homing Human Injection of therapeutic agent Intravenous Left Light Mediator of activation protein Medicine Mesenchymal Stem Cells Methods Modeling Modification Molecular Mus Mutation Nature Neurodegenerative Disorders Pathology Patients Photoreceptors Play Process Public Health Rattus Research Research Proposals Retina Retinal Retinal Degeneration Risk Rodent Model Role Route Running Safety Social Impacts Staging Stem cells Stromal Cell-Derived Factor 1 Structure Surgeon Testing Therapeutic Effect Time Translations Treatment Protocols Vision autocrine base chemokine college cytokine effective therapy experience gene therapy implantation intravenous administration intravenous injection intravitreal injection lens novel strategies paracrine photoreceptor degeneration protective effect regenerative repaired response stem cell biology stem cell therapy subretinal injection

项目摘要

Summary Retinal degeneration and related diseases are the leading cause of blindness and represent a major public health burden with economical and social impacts. As photoreceptor loss, the development of secondary vascular pathology causes disastrous consequences for vision. There is no effective treatment available. Our recent study revealed that a single intravenous injection of bone marrow derived mesenchymal stem cells (MSCs) at early stages of degeneration can preserve photoreceptors from death, sustain visual function, and limit pathological vascular changes in a rodent model of retinal degeneration. We propose to develop a treatment protocol that preserves vision and limits vascular pathology using non-invasive stem cell therapy in rodent models for retinal degeneration. We hypothesize that systemic administration of MSCs to treat ongoing retinal degeneration will slow the progress of photoreceptor loss and stabilize/repair the secondary vascular pathology by promoting the release of paracrine and autocrine mediators. The following specific aims are proposed: (1) Determine dose-response and long-term safety and efficacy of MSC treatment at early stages of degeneration in the RCS rat; (2) Investigate the neuro-vascular protective effects of MSCs at later stages of degeneration in the RCS rat and in Elovl4 mouse; (3) Examine the molecular mechanism of MSC homing to the retina and efficacy after intravenous administration. Based on the current extensive clinical experience using MSCs as therapy for both regenerative and degenerative medicine, if positive results are obtained in animal models, this treatment has a realistic likelihood of translation to the clinic.

总结