Heikkilä P, Tryggvason K, Thorner P
Division of Matrix Biology, Department of Medical Biochemistry and Biophysics, Karolinska Institute, Stockholm, Sweden.
Exp Nephrol. 2000 Jan-Feb;8(1):1-7. doi: 10.1159/000020641.
Several animal models for Alport syndrome have been described. These are available for studies on the pathogenetic mechanisms of the disease, as well as for the development of new technologies for gene therapy in this progressive hereditary kidney disease. This review summarizes current knowledge on the molecular basis of Alport syndrome, and on the animal models which all remarkably well resemble the human disease. Recent work aimed at the development of gene therapy, including hurdles and progress are discussed.
已经描述了几种阿尔波特综合征的动物模型。这些模型可用于研究该疾病的发病机制，以及开发针对这种进行性遗传性肾病的基因治疗新技术。本综述总结了关于阿尔波特综合征分子基础以及与人类疾病非常相似的动物模型的现有知识。讨论了近期旨在开发基因治疗的工作，包括遇到的障碍和取得的进展。
