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2020 5
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Page 1
Krabbe disease: New hope for an old disease.
Bradbury AM, Bongarzone ER, Sands MS. Bradbury AM, et al. Neurosci Lett. 2021 May 1;752:135841. doi: 10.1016/j.neulet.2021.135841. Epub 2021 Mar 22. Neurosci Lett. 2021. PMID: 33766733 Free PMC article. Review.
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. Sci Rep. 2021 Jul 14;11(1):14486. doi: 10.1038/s41598-021-93601-1. Sci Rep. 2021. PMID: 34262084 Free PMC article.
Cell-autonomous expression of the acid hydrolase galactocerebrosidase.
Mikulka CR, Dearborn JT, Benitez BA, Strickland A, Liu L, Milbrandt J, Sands MS. Mikulka CR, et al. Proc Natl Acad Sci U S A. 2020 Apr 21;117(16):9032-9041. doi: 10.1073/pnas.1917675117. Epub 2020 Apr 6. Proc Natl Acad Sci U S A. 2020. PMID: 32253319 Free PMC article.
Macrophage secretion of miR-106b-5p causes renin-dependent hypertension.
Oh J, Matkovich SJ, Riek AE, Bindom SM, Shao JS, Head RD, Barve RA, Sands MS, Carmeliet G, Osei-Owusu P, Knutsen RH, Zhang H, Blumer KJ, Nichols CG, Mecham RP, Baldán Á, Benitez BA, Sequeira-Lopez ML, Gomez RA, Bernal-Mizrachi C. Oh J, et al. Nat Commun. 2020 Sep 23;11(1):4798. doi: 10.1038/s41467-020-18538-x. Nat Commun. 2020. PMID: 32968066 Free PMC article.
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, Shelton GD, Bongarzone ER, Gray SJ, Vite CH. Bradbury AM, et al. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
19 results