Evolving therapies in neuronopathic LSDs: opportunities and challenges
Author:
Publisher
Springer Science and Business Media LLC
Subject
Cellular and Molecular Neuroscience,Neurology (clinical),Biochemistry
Link
https://link.springer.com/content/pdf/10.1007/s11011-022-00939-0.pdf
Reference86 articles.
1. Ballabio A, Bonifacino JS (2020) ‘Lysosomes as dynamic regulators of cell and organismal homeostasis’. Nat Rev Mol Cell Biol 21:101–118
2. Barker JN, Weisdorf DJ, Wagner JE (2001) ‘Creation of a double chimera after the transplantation of umbilical-cord blood from two partially matched unrelated donors’. N Engl J Med 344:1870–1871
3. Barton NW, Brady RO, Dambrosia JM, Di Bisceglie AM, Doppelt SH, Hill SC, Mankin HJ, Murray GJ, Parker RI, Argoff CE et al (1991) ‘Replacement therapy for inherited enzyme deficiency–macrophage-targeted glucocerebrosidase for Gaucher’s disease’. N Engl J Med 324:1464–1470
4. Barton NW, Furbish FS, Murray GJ, Garfield M, Brady RO (1990) ‘Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease’. Proc Natl Acad Sci U S A 87:1913–1916
5. Bellettato CM, Scarpa M (2018) ‘Possible strategies to cross the blood-brain barrier’. Ital J Pediatr 44:131
Cited by 4 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. Hurler Syndrome Glycosaminoglycans Decrease in Cerebrospinal Fluid without Brain‐Targeted Therapy;Annals of Neurology;2023-09-23
2. Gene therapy for lysosomal storage diseases: Current clinical trial prospects;Frontiers in Genetics;2023-01-13
3. Sanfilippo Syndrome: Optimizing Care with a Multidisciplinary Approach;Journal of Multidisciplinary Healthcare;2022-09
4. Increasing precision in the measurement of change in pediatric neurodegenerative disease;Molecular Genetics and Metabolism;2022-09
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