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

Year Number of Results
2002 3
2003 3
2004 1
2005 4
2006 4
2007 1
2008 6
2009 8
2010 8
2011 10
2012 7
2013 8
2014 11
2015 10
2016 7
2017 9
2018 1
2019 5
2020 3
2021 3
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99 results
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Page 1
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. Among authors: kaspar bk. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Free article. Clinical Trial.
Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS.
Bravo-Hernandez M, Tadokoro T, Navarro MR, Platoshyn O, Kobayashi Y, Marsala S, Miyanohara A, Juhas S, Juhasova J, Skalnikova H, Tomori Z, Vanicky I, Studenovska H, Proks V, Chen P, Govea-Perez N, Ditsworth D, Ciacci JD, Gao S, Zhu W, Ahrens ET, Driscoll SP, Glenn TD, McAlonis-Downes M, Da Cruz S, Pfaff SL, Kaspar BK, Cleveland DW, Marsala M. Bravo-Hernandez M, et al. Among authors: kaspar bk. Nat Med. 2020 Jan;26(1):118-130. doi: 10.1038/s41591-019-0674-1. Epub 2019 Dec 23. Nat Med. 2020. PMID: 31873312 Free PMC article.
Intravenous injections in neonatal mice.
Gombash Lampe SE, Kaspar BK, Foust KD. Gombash Lampe SE, et al. Among authors: kaspar bk. J Vis Exp. 2014 Nov 11;(93):e52037. doi: 10.3791/52037. J Vis Exp. 2014. PMID: 25407048 Free PMC article.
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Cain JT, Likhite S, White KA, Timm DJ, Davis SS, Johnson TB, Dennys-Rivers CN, Rinaldi F, Motti D, Corcoran S, Morales P, Pierson C, Hughes SM, Lee SY, Kaspar BK, Meyer K, Weimer JM. Cain JT, et al. Among authors: kaspar bk. Mol Ther. 2019 Oct 2;27(10):1836-1847. doi: 10.1016/j.ymthe.2019.06.015. Epub 2019 Jul 10. Mol Ther. 2019. PMID: 31331814 Free PMC article.
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Meyer K, et al. Among authors: kaspar bk. Mol Ther. 2015 Mar;23(3):477-87. doi: 10.1038/mt.2014.210. Epub 2014 Oct 31. Mol Ther. 2015. PMID: 25358252 Free PMC article.
A large animal model of spinal muscular atrophy and correction of phenotype.
Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH. Duque SI, et al. Among authors: kaspar bk. Ann Neurol. 2015 Mar;77(3):399-414. doi: 10.1002/ana.24332. Epub 2015 Feb 9. Ann Neurol. 2015. PMID: 25516063 Free PMC article.
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