首页 | 本学科首页   官方微博 | 高级检索  
     


Gene therapy for lysosomal storage diseases.
Authors:Mark S Sands  Beverly L Davidson
Affiliation:Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO 63110, USA. msands@im.wustl.edu
Abstract:Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders with complex clinical phenotypes that include both systemic and central nervous system pathologies. In recent years, the identification or development of mouse models recapitulating the clinical course of the LSDs has been instrumental in evaluating therapeutic strategies. Here, we review the various gene replacement strategies for target organs affected in many LSDs and describe briefly the various vector systems employed to test how best to accomplish long-lasting therapies for these fatal disorders.
Keywords:
设为首页 | 免责声明 | 关于勤云 | 加入收藏

Copyright©北京勤云科技发展有限公司  京ICP备09084417号