Gene therapy progress and prospects: gene therapy of lysosomal storage disorders
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.nature.com/articles/3302092.pdf
Reference50 articles.
1. Suzuki K . Lysosomal diseases. In: Graham DI, Lantos PL (eds) Greenfield's Neuropathology. Arnold: London, 2002, pp 653–735.
2. Wraith JE . Advances in the treatment of lysosomal storage disease. Dev Med Child Neurol 2001; 43: 639–646.
3. Weinreb NJ et al. Effectiveness of enzyme replacement therapy in 1028 patients with Type I Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher registry. Am J Med 2002; 113: 112–119.
4. Eng CM et al. Safety and efficacy of recombinant human α-galactosidase A replacement in Fabry disease. N Engl J Med 2001; 345: 9–16.
5. Schiffman R et al. Enzyme replacement therapy in Fabry disease: a randomised controlled trial. JAMA 2001; 285: 2743–2749.
Cited by 97 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Lysosomal Dysfunction: Connecting the Dots in the Landscape of Human Diseases;Biology;2024-01-07
2. Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders;Advanced Drug Delivery Reviews;2023-06
3. IgG Fusion Proteins for Brain Delivery of Biologics via Blood–Brain Barrier Receptor-Mediated Transport;Pharmaceutics;2022-07-15
4. Delineating the Neuropathology of Lysosomal Storage Diseases Using Patient-Derived Induced Pluripotent Stem Cells;Stem Cells and Development;2022-05-01
5. The surgical management of spinal disorders in lysosomal storage diseases: a systematic review;ANZ Journal of Surgery;2022-01-04
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3