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Table representation of search results timeline featuring number of search results per year.

Year Number of Results
2013 4
2014 2
2015 2
2016 2
2018 2
2019 2
2020 4
2021 0
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14 results
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Page 1
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. Among authors: bagel jh. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease).
Bradbury AM, Rafi MA, Bagel JH, Brisson BK, Marshall MS, Pesayco Salvador J, Jiang X, Swain GP, Prociuk ML, ODonnell PA, Fitzgerald C, Ory DS, Bongarzone ER, Shelton GD, Wenger DA, Vite CH. Bradbury AM, et al. Among authors: bagel jh. Hum Gene Ther. 2018 Jul;29(7):785-801. doi: 10.1089/hum.2017.151. Epub 2018 Mar 14. Hum Gene Ther. 2018. PMID: 29316812 Free PMC article.
Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling.
Fan M, Sidhu R, Fujiwara H, Tortelli B, Zhang J, Davidson C, Walkley SU, Bagel JH, Vite C, Yanjanin NM, Porter FD, Schaffer JE, Ory DS. Fan M, et al. Among authors: bagel jh. J Lipid Res. 2013 Oct;54(10):2800-14. doi: 10.1194/jlr.M040618. Epub 2013 Jul 23. J Lipid Res. 2013. PMID: 23881911 Free PMC article.
Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease.
Vite CH, Bagel JH, Swain GP, Prociuk M, Sikora TU, Stein VM, O'Donnell P, Ruane T, Ward S, Crooks A, Li S, Mauldin E, Stellar S, De Meulder M, Kao ML, Ory DS, Davidson C, Vanier MT, Walkley SU. Vite CH, et al. Among authors: bagel jh. Sci Transl Med. 2015 Feb 25;7(276):276ra26. doi: 10.1126/scitranslmed.3010101. Sci Transl Med. 2015. PMID: 25717099 Free PMC article.
Hypervitaminosis D in guinea pigs with α-mannosidosis.
Jensen JA, Brice AK, Bagel JH, Mexas AM, Yoon SY, Wolfe JH. Jensen JA, et al. Among authors: bagel jh. Comp Med. 2013 Apr;63(2):156-62. Comp Med. 2013. PMID: 23582422 Free PMC article.
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