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.
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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
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