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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: chandra s. 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.
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood.
Zhang L, Yates B, Murphy R, Liu S, Xie L, Handyside B, Sihn CR, Bouwman T, Galicia N, Tan D, Fonck C, Arens J, Clark A, Zhang W, Chandra S, Srimani J, Holcomb J, Van Tuyl A, Henshaw J, Vettermann C, Siso S, Su C, Bullens S, Bunting S, O'Neill C, Fong S. Zhang L, et al. Among authors: chandra s. Mol Ther Methods Clin Dev. 2022 Aug 13;26:519-531. doi: 10.1016/j.omtm.2022.08.002. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 36092364 Free PMC article.
Safety, pharmacokinetics and CNS distribution of tralesinidase alfa administered via intracerebroventricular infusion to juvenile cynomolgus monkeys.
Pinkstaff J, McCullagh E, Grover A, Melton AC, Cherukuri A, Wait JC, Nguyen A, Butt MT, Trombley JL, Reed RP, Adams EL, Boyd RB, Chandra S, Henshaw J, O'Neill CA, Zanelli E, Kovalchin J. Pinkstaff J, et al. Among authors: chandra s. Toxicol Rep. 2023 Mar 1;10:357-366. doi: 10.1016/j.toxrep.2023.02.014. eCollection 2023. Toxicol Rep. 2023. PMID: 36923444 Free PMC article.
Brain Alterations in Aged OVT73 Sheep Model of Huntington's Disease: An MRI Based Approach.
Taghian T, Gallagher J, Batcho E, Pullan C, Kuchel T, Denney T, Perumal R, Moore S, Muirhead R, Herde P, Johns D, Christou C, Taylor A, Passler T, Pulaparthi S, Hall E, Chandra S, O'Neill CA, Gray-Edwards H. Taghian T, et al. Among authors: chandra s. J Huntingtons Dis. 2022;11(4):391-406. doi: 10.3233/JHD-220526. J Huntingtons Dis. 2022. PMID: 36189602 Free PMC article.
Natural history of Tay-Sachs disease in sheep.
Story B, Taghian T, Gallagher J, Koehler J, Taylor A, Randle A, Nielsen K, Gross A, Maguire A, Carl S, Johnson S, Fernau D, Diffie E, Cuddon P, Corado C, Chandra S, Sena-Esteves M, Kolodny E, Jiang X, Martin D, Gray-Edwards H. Story B, et al. Among authors: chandra s. Mol Genet Metab. 2021 Sep-Oct;134(1-2):164-174. doi: 10.1016/j.ymgme.2021.08.009. Epub 2021 Aug 21. Mol Genet Metab. 2021. PMID: 34456134 Free PMC article.
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
Babcock MC, Mikulka CR, Wang B, Chandriani S, Chandra S, Xu Y, Webster K, Feng Y, Nelvagal HR, Giaramita A, Yip BK, Lo M, Jiang X, Chao Q, Woloszynek JC, Shen Y, Bhagwat S, Sands MS, Crawford BE. Babcock MC, et al. Among authors: chandra s. Sci Rep. 2021 Jul 14;11(1):14486. doi: 10.1038/s41598-021-93601-1. Sci Rep. 2021. PMID: 34262084 Free PMC article.
2,783 results