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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: schmelzer l. 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: schmelzer l. 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.
Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.
Rodino-Klapac LR, Janssen PM, Shontz KM, Canan B, Montgomery CL, Griffin D, Heller K, Schmelzer L, Handy C, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Rodino-Klapac LR, et al. Among authors: schmelzer l. Hum Mol Genet. 2013 Dec 15;22(24):4929-37. doi: 10.1093/hmg/ddt342. Epub 2013 Jul 17. Hum Mol Genet. 2013. PMID: 23863459 Free PMC article.
Molecular age prediction using skull bone samples from individuals with and without signs of decomposition: a multivariate approach combining analysis of posttranslational protein modifications and DNA methylation.
Becker J, Bühren V, Schmelzer L, Reckert A, Eickhoff SB, Ritz S, Naue J. Becker J, et al. Among authors: schmelzer l. Int J Legal Med. 2024 Sep 11. doi: 10.1007/s00414-024-03314-z. Online ahead of print. Int J Legal Med. 2024. PMID: 39256256
28 results