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The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
Batra R, Nelles DA, Roth DM, Krach F, Nutter CA, Tadokoro T, Thomas JD, Sznajder ŁJ, Blue SM, Gutierrez HL, Liu P, Aigner S, Platoshyn O, Miyanohara A, Marsala M, Swanson MS, Yeo GW. Batra R, et al. Among authors: marsala m. Nat Biomed Eng. 2021 Feb;5(2):157-168. doi: 10.1038/s41551-020-00607-7. Epub 2020 Sep 14. Nat Biomed Eng. 2021. PMID: 32929188 Free PMC article.
Combinational spinal GAD65 gene delivery and systemic GABA-mimetic treatment for modulation of spasticity.
Kakinohana O, Hefferan MP, Miyanohara A, Nejime T, Marsala S, Juhas S, Juhasova J, Motlik J, Kucharova K, Strnadel J, Platoshyn O, Lazar P, Galik J, Vinay L, Marsala M. Kakinohana O, et al. Among authors: marsala s, marsala m. PLoS One. 2012;7(1):e30561. doi: 10.1371/journal.pone.0030561. Epub 2012 Jan 23. PLoS One. 2012. PMID: 22291989 Free PMC article.
Survival and differentiation of human embryonic stem cell-derived neural precursors grafted spinally in spinal ischemia-injured rats or in naive immunosuppressed minipigs: a qualitative and quantitative study.
Kakinohana O, Juhasova J, Juhas S, Motlik J, Platoshyn O, Galik J, Hefferan M, Yuan SH, Vidal JG, Carson CT, van Gorp S, Goldberg D, Leerink M, Lazar P, Marsala S, Miyanohara A, Keshavarzi S, Ciacci JD, Marsala M. Kakinohana O, et al. Among authors: marsala s, marsala m. Cell Transplant. 2012;21(12):2603-19. doi: 10.3727/096368912X653200. Epub 2012 Aug 10. Cell Transplant. 2012. PMID: 22889456 Free article.
ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43.
Arnold ES, Ling SC, Huelga SC, Lagier-Tourenne C, Polymenidou M, Ditsworth D, Kordasiewicz HB, McAlonis-Downes M, Platoshyn O, Parone PA, Da Cruz S, Clutario KM, Swing D, Tessarollo L, Marsala M, Shaw CE, Yeo GW, Cleveland DW. Arnold ES, et al. Among authors: marsala m. Proc Natl Acad Sci U S A. 2013 Feb 19;110(8):E736-45. doi: 10.1073/pnas.1222809110. Epub 2013 Feb 4. Proc Natl Acad Sci U S A. 2013. PMID: 23382207 Free PMC article.
Human mutant huntingtin disrupts vocal learning in transgenic songbirds.
Liu WC, Kohn J, Szwed SK, Pariser E, Sepe S, Haripal B, Oshimori N, Marsala M, Miyanohara A, Lee R. Liu WC, et al. Among authors: marsala m. Nat Neurosci. 2015 Nov;18(11):1617-22. doi: 10.1038/nn.4133. Epub 2015 Oct 5. Nat Neurosci. 2015. PMID: 26436900
Thoracic 9 Spinal Transection-Induced Model of Muscle Spasticity in the Rat: A Systematic Electrophysiological and Histopathological Characterization.
Corleto JA, Bravo-Hernández M, Kamizato K, Kakinohana O, Santucci C, Navarro MR, Platoshyn O, Cizkova D, Lukacova N, Taylor J, Marsala M. Corleto JA, et al. Among authors: marsala m. PLoS One. 2015 Dec 29;10(12):e0144642. doi: 10.1371/journal.pone.0144642. eCollection 2015. PLoS One. 2015. PMID: 26713446 Free PMC article.
Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs.
Jiang J, Zhu Q, Gendron TF, Saberi S, McAlonis-Downes M, Seelman A, Stauffer JE, Jafar-Nejad P, Drenner K, Schulte D, Chun S, Sun S, Ling SC, Myers B, Engelhardt J, Katz M, Baughn M, Platoshyn O, Marsala M, Watt A, Heyser CJ, Ard MC, De Muynck L, Daughrity LM, Swing DA, Tessarollo L, Jung CJ, Delpoux A, Utzschneider DT, Hedrick SM, de Jong PJ, Edbauer D, Van Damme P, Petrucelli L, Shaw CE, Bennett CF, Da Cruz S, Ravits J, Rigo F, Cleveland DW, Lagier-Tourenne C. Jiang J, et al. Among authors: marsala m. Neuron. 2016 May 4;90(3):535-50. doi: 10.1016/j.neuron.2016.04.006. Epub 2016 Apr 21. Neuron. 2016. PMID: 27112497 Free PMC article.
205 results