How close are we to therapies for Sanfilippo disease?
Author:
Funder
Fundacja Sanfilippo
Publisher
Springer Science and Business Media LLC
Subject
Cellular and Molecular Neuroscience,Clinical Neurology,Biochemistry
Link
http://link.springer.com/article/10.1007/s11011-017-0111-4/fulltext.html
Reference49 articles.
1. Aldenhoven M, Jones SA, Bonney D, Borrill RE, Coussons M, Mercer J, Bierings MB, Versluys B, van Hasselt PM, Wijburg FA, van der Ploeg AT, Wynn RF, Boelens JJ (2015) Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines. Biol Blood Marrow Transplant 21:1106–1109. https://doi.org/10.1016/j.bbmt.2015.02.011
2. Andrade F, Aldámiz-Echevarría L, Llarena M, Couce ML (2015) Sanfilippo syndrome: overall review. Pediatr Int 57:331–338. https://doi.org/10.1111/ped.12636
3. Aoyagi-Scharber M, Crippen-Harmon D, Lawrence R, Vincelette J, Yogalingam G, Prill H, Yip BK, Baridon B, Vitelli C, Lee A, Gorostiza O, Adintori EG, Minto WC, Van Vleet JL, Yates B, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Fitzpatrick PA, LeBowitz JH, Bullens S, Crawford BE, Bunting S (2017) Clearance of heparan sulfate and attenuation of CNS pathology by intracerebroventricular BMN 250 in Sanfilippo type B mice. Mol Ther Methods Clin Dev 6:43–53. https://doi.org/10.1016/j.omtm.2017.05.009
4. Beard H, Luck AJ, Hassiotis S, King B, Trim PJ, Snel MF, Hopwood JJ, Hemsley KM (2015) Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice. Mol Genet Metab 115:33–40. https://doi.org/10.1016/j.ymgme.2015.03.002
5. Boado RJ, Lu JZ, Hui EK, Pardridge WM (2014) Insulin receptor antibody-sulfamidase fusion protein penetrates the primate blood-brain barrier and reduces glycosoaminoglycans in Sanfilippo type A cells. Mol Pharm 11:2928–2934. https://doi.org/10.1021/mp500258p
Cited by 49 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. An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome);Scientific Reports;2023-10-27
3. Genistein - characterization of the molecule, similarity to estradiol and pharmacological potential;Acta Poloniae Pharmaceutica - Drug Research;2023-06-29
4. Activities of (Poly)phenolic Antioxidants and Other Natural Autophagy Modulators in the Treatment of Sanfilippo Disease: Remarkable Efficacy of Resveratrol in Cellular and Animal Models;Neurotherapeutics;2022-11-07
5. Sanfilippo Syndrome: Optimizing Care with a Multidisciplinary Approach;Journal of Multidisciplinary Healthcare;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