Department of Ophthalmology, Moran Eye Center, University of Utah, Salt Lake City, Utah 84132, USA.
J Comp Neurol. 2011 Oct 1;519(14):2713-33. doi: 10.1002/cne.22703.
Retinitis pigmentosa (RP) is an inherited blinding disease characterized by progressive loss of retinal photoreceptors. There are numerous rodent models of retinal degeneration, but most are poor platforms for interventions that will translate into clinical practice. The rabbit possesses a number of desirable qualities for a model of retinal disease including a large eye and an existing and substantial knowledge base in retinal circuitry, anatomy, and ophthalmology. We have analyzed degeneration, remodeling, and reprogramming in a rabbit model of retinal degeneration, expressing a rhodopsin proline 347 to leucine transgene in a TgP347L rabbit as a powerful model to study the pathophysiology and treatment of retinal degeneration. We show that disease progression in the TgP347L rabbit closely tracks human cone-sparing RP, including the cone-associated preservation of bipolar cell signaling and triggering of reprogramming. The relatively fast disease progression makes the TgP347L rabbit an excellent model for gene therapy, cell biological intervention, progenitor cell transplantation, surgical interventions, and bionic prosthetic studies.
色素性视网膜炎（RP）是一种遗传性致盲疾病，其特征是视网膜光感受器逐渐丧失。有许多用于视网膜变性的啮齿动物模型，但大多数模型都不适合转化为临床实践的干预措施。兔子在视网膜疾病模型方面具有许多理想的特性，包括大眼球和现有的、大量的视网膜回路、解剖学和眼科学知识库。我们已经分析了视网膜变性模型中的变性、重塑和重编程，在 TgP347L 兔中表达视紫红质脯氨酸 347 到亮氨酸转基因，作为研究视网膜变性的病理生理学和治疗的强大模型。我们表明，TgP347L 兔的疾病进展与人类的 cone-sparing RP 密切相关，包括与双极细胞信号相关的 cone 保留和重编程的触发。相对较快的疾病进展使 TgP347L 兔成为基因治疗、细胞生物学干预、祖细胞移植、手术干预和仿生假体研究的优秀模型。
