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Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.
Ellinwood NM, Valentine BN, Hess AS, Jens JK, Snella EM, Jamil M, Hostetter SJ, Jeffery ND, Smith JD, Millman ST, Parsons RL, Butt MT, Chandra S, Egeland MT, Assis AB, Nelvagal HR, Cooper JD, Nestrasil I, Mueller BA, Labounek R, Paulson A, Prill H, Liu XY, Zhou H, Lawrence R, Crawford BE, Grover A, Cherala G, Melton AC, Cherukuri A, Vuillemenot BR, Wait JCM, O'Neill CA, Pinkstaff J, Kovalchin J, Zanelli E, McCullagh E. Ellinwood NM, et al. Among authors: lawrence r. J Pharmacol Exp Ther. 2022 Sep;382(3):277-286. doi: 10.1124/jpet.122.001119. Epub 2022 Jun 18. J Pharmacol Exp Ther. 2022. PMID: 35717448 Free PMC article.
Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I.
Wang RY, Aminian A, McEntee MF, Kan SH, Simonaro CM, Lamanna WC, Lawrence R, Ellinwood NM, Guerra C, Le SQ, Dickson PI, Esko JD. Wang RY, et al. Among authors: lawrence r. Mol Genet Metab. 2014 Aug;112(4):286-93. doi: 10.1016/j.ymgme.2014.05.015. Epub 2014 Jun 6. Mol Genet Metab. 2014. PMID: 24951454 Free PMC article.
BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.
Yogalingam G, Luu AR, Prill H, Lo MJ, Yip B, Holtzinger J, Christianson T, Aoyagi-Scharber M, Lawrence R, Crawford BE, LeBowitz JH. Yogalingam G, et al. Among authors: lawrence r. PLoS One. 2019 Jan 18;14(1):e0207836. doi: 10.1371/journal.pone.0207836. eCollection 2019. PLoS One. 2019. PMID: 30657762 Free PMC article.
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
Grover A, Crippen-Harmon D, Nave L, Vincelette J, Wait JCM, Melton AC, Lawrence R, Brown JR, Webster KA, Yip BK, Baridon B, Vitelli C, Rigney S, Christianson TM, Tiger PMN, Lo MJ, Holtzinger J, Shaywitz AJ, Crawford BE, Fitzpatrick PA, LeBowitz JH, Bullens S, Aoyagi-Scharber M, Bunting S, O'Neill CA, Pinkstaff J, Bagri A. Grover A, et al. Among authors: lawrence r. Drug Deliv Transl Res. 2020 Apr;10(2):425-439. doi: 10.1007/s13346-019-00683-6. Drug Deliv Transl Res. 2020. PMID: 31942701 Free PMC article.
Glycan-based biomarkers for mucopolysaccharidoses.
Lawrence R, Brown JR, Lorey F, Dickson PI, Crawford BE, Esko JD. Lawrence R, et al. Mol Genet Metab. 2014 Feb;111(2):73-83. doi: 10.1016/j.ymgme.2013.07.016. Epub 2013 Jul 29. Mol Genet Metab. 2014. PMID: 23958290 Free PMC article. Review.
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.
Kan SH, Aoyagi-Scharber M, Le SQ, Vincelette J, Ohmi K, Bullens S, Wendt DJ, Christianson TM, Tiger PM, Brown JR, Lawrence R, Yip BK, Holtzinger J, Bagri A, Crippen-Harmon D, Vondrak KN, Chen Z, Hague CM, Woloszynek JC, Cheung DS, Webster KA, Adintori EG, Lo MJ, Wong W, Fitzpatrick PA, LeBowitz JH, Crawford BE, Bunting S, Dickson PI, Neufeld EF. Kan SH, et al. Among authors: lawrence r. Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14870-5. doi: 10.1073/pnas.1416660111. Epub 2014 Sep 29. Proc Natl Acad Sci U S A. 2014. PMID: 25267636 Free PMC article.
1,656 results