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Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
Author / Creator:Thomsen, Gretchen ; Burghes, Arthur H. M. ; Hsieh, Caroline ; Do, Janet ; Chu, Binh T. T. ; Perry, Stephanie ; Barkho, Basam ; Kaufmann, Petra ; Sproule, Douglas M. ; Feltner, Douglas E. ; Chung, Wendy K. ; McGovern, Vicki L. ; Hevner, Robert F. ; Conces, Miriam ; Pierson, Christopher R. ; Scoto, Mariacristina ; Muntoni, Francesco ; Mendell, Jerry R. ; Foust, Kevin D.
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2021-10, Vol.27 (10), p.1701-1711
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Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study
Author / Creator:Crawford, Thomas O ; Paushkin, Sergey V ; Kobayashi, Dione T ; Forrest, Suzanne J ; Joyce, Cynthia L ; Finkel, Richard S ; Kaufmann, Petra ; Swoboda, Kathryn J ; Tiziano, Danilo ; Lomastro, Rosa ; Li, Rebecca H ; Trachtenberg, Felicia L ; Plasterer, Thomas ; Chen, Karen S
Publisher:United States: Public Library of Science
Journal title:PloS one, 2012-04, Vol.7 (4), p.e33572-e33572
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Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs
Author / Creator:Poirier, Agnès ; Weetall, Marla ; Heinig, Katja ; Bucheli, Franz ; Schoenlein, Kerstin ; Alsenz, Jochem ; Bassett, Simon ; Ullah, Mohammed ; Senn, Claudia ; Ratni, Hasane ; Naryshkin, Nikolai ; Paushkin, Sergey ; Mueller, Lutz
Publisher:United States: John Wiley & Sons, Inc
Journal title:Pharmacology research & perspectives, 2018-12, Vol.6 (6), p.e00447-n/a
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SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials
Author / Creator:Zaworski, Phillip ; von Herrmann, Katharine M ; Taylor, Shannon ; Sunshine, Sara S ; McCarthy, Kathleen ; Risher, Nicole ; Newcomb, Tara ; Weetall, Marla ; Prior, Thomas W ; Swoboda, Kathryn J ; Chen, Karen S ; Paushkin, Sergey
Publisher:United States: Public Library of Science
Journal title:PloS one, 2016-03, Vol.11 (3), p.e0150640-e0150640
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Survival Motor Neuron (SMN) protein is required for normal mouse liver development
Author / Creator:Szunyogova, Eva ; Zhou, Haiyan ; Maxwell, Gillian K. ; Powis, Rachael A. ; Muntoni, Francesco ; Gillingwater, Thomas H. ; Parson, Simon H.
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2016-10, Vol.6 (1), p.34635-34635, Article 34635
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Analysis of SMN protein in umbilical cord blood and postnatal peripheral blood of neonates with SMA: a rationale for prompt treatment initiation to prevent SMA development
Author / Creator:Otsuki, Noriko ; Kato, Tamaki ; Yokomura, Mamoru ; Urano, Mari ; Matsuo, Mari ; Kobayashi, Emiko ; Haginoya, Kazuhiro ; Awano, Hiroyuki ; Takeshima, Yasuhiro ; Saito, Toshio ; Saito, Kayoko
Publisher:England: BioMed Central Ltd
Journal title:Orphanet journal of rare diseases, 2025-02, Vol.20 (1), p.91-14, Article 91
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The phospho-landscape of the survival of motoneuron protein (SMN) protein: relevance for spinal muscular atrophy (SMA)
Author / Creator:Detering, Nora Tula ; Schüning, Tobias ; Hensel, Niko ; Claus, Peter
Publisher:Cham: Springer International Publishing
Journal title:Cellular and molecular life sciences : CMLS, 2022-09, Vol.79 (9), p.497-497, Article 497
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LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts
Author / Creator:Touznik, Aleksander ; Maruyama, Rika ; Hosoki, Kana ; Echigoya, Yusuke ; Yokota, Toshifumi
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2017-06, Vol.7 (1), p.3672-9, Article 3672
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SMN protein promotes membrane compartmentalization of ribosomal protein S6 transcript in human fibroblasts
Author / Creator:Gabanella, Francesca ; Onori, Annalisa ; Ralli, Massimo ; Greco, Antonio ; Passananti, Claudio ; Di Certo, Maria Grazia
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2020-11, Vol.10 (1), p.19000-19000, Article 19000
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Depletion of SMN protein in mesenchymal progenitors impairs the development of bone and neuromuscular junction in spinal muscular atrophy
Author / Creator:Hann, Sang-Hyeon ; Kim, Seon-Yong ; Kim, Ye Lynne ; Jo, Young-Woo ; Kang, Jong-Seol ; Park, Hyerim ; Choi, Se-Young ; Kong, Young-Yun
Publisher:England: eLife Sciences Publications Ltd
Journal title:eLife, 2024-02, Vol.12
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ArticlesPeer ReviewedFull Text
Depletion of SMN protein in mesenchymal progenitors impairs the development of bone and neuromuscular junction in spinal muscular atrophy
Author / Creator:Hann, Sang-Hyeon ; Kim, Seon-Yong ; Kim, Ye Lynne ; Jo, Young-Woo ; Kang, Jong-Seol ; Park, Hyerim ; Choi, Se-Young ; Kong, Young-Yun
Journal title:eLife, 2024-02, Vol.12
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A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy Controls
Author / Creator:Wadman, Renske I ; Stam, Marloes ; Jansen, Marc D ; van der Weegen, Yana ; Wijngaarde, Camiel A ; Harschnitz, Oliver ; Sodaar, Peter ; Braun, Kees P J ; Dooijes, Dennis ; Lemmink, Henny H ; van den Berg, Leonard H ; van der Pol, W Ludo
Publisher:United States: Public Library of Science
Journal title:PloS one, 2016-11, Vol.11 (11), p.e0167087-e0167087
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Recombinant SMN protein synergizes with spinal muscular atrophy therapy to counteract pathological motor neuron phenotypes
Author / Creator:Brambilla, Liliana ; Valori, Chiara F ; Guidotti, Giulia ; Martorana, Francesca ; Sulmona, Claudia ; De Martini, Lisa Benedetta ; Canciani, Anselmo ; Fumagalli, Marco ; Talpo, Francesca ; Biella, Gerardo ; Di Pasquale, Elisa ; Iacobucci, Claudio ; Forneris, Federico ; Zhou, Haiyan ; Rossi, Daniela
Publisher:England: BioMed Central
Journal title:Translational neurodegeneration, 2024-12, Vol.13 (1), p.63-5, Article 63
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Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy
Author / Creator:Niba, Emma Tabe Eko ; Nishio, Hisahide ; Wijaya, Yogik Onky Silvana ; Ar Rochmah, Mawaddah ; Takarada, Toru ; Takeuchi, Atsuko ; Kimizu, Tomokazu ; Okamoto, Kentaro ; Saito, Toshio ; Awano, Hiroyuki ; Takeshima, Yasuhiro ; Shinohara, Masakazu
Publisher:Switzerland: MDPI AG
Journal title:Genes, 2022-01, Vol.13 (2), p.205
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Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug‐like compounds
Author / Creator:Cherry, Jonathan J. ; Osman, Erkan Y. ; Evans, Matthew C. ; Choi, Sungwoon ; Xing, Xuechao ; Cuny, Gregory D. ; Glicksman, Marcie A. ; Lorson, Christian L. ; Androphy, Elliot J.
Publisher:London: Nature Publishing Group UK
Journal title:EMBO molecular medicine, 2013-07, Vol.5 (7), p.1103-1118
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Calpain Inhibition Increases SMN Protein in Spinal Cord Motoneurons and Ameliorates the Spinal Muscular Atrophy Phenotype in Mice
Author / Creator:de la Fuente, Sandra ; Sansa, Alba ; Periyakaruppiah, Ambika ; Garcera, Ana ; Soler, Rosa M.
Publisher:New York: Springer US
Journal title:Molecular neurobiology, 2019-06, Vol.56 (6), p.4414-4427
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Different Stability and Proteasome-Mediated Degradation Rate of SMN Protein Isoforms
Author / Creator:Locatelli, Denise ; Terao, Mineko ; Kurosaki, Mami ; Zanellati, Maria Clara ; Pletto, Daniela Rita ; Finardi, Adele ; Colciaghi, Francesca ; Garattini, Enrico ; Battaglia, Giorgio Stefano
Publisher:United States: Public Library of Science
Journal title:PloS one, 2015-07, Vol.10 (7), p.e0134163-e0134163
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Small-molecule flunarizine increases SMN protein in nuclear Cajal bodies and motor function in a mouse model of spinal muscular atrophy
Author / Creator:Sapaly, Delphine ; Dos Santos, Matthieu ; Delers, Perrine ; Biondi, Olivier ; Quérol, Gwendoline ; Houdebine, Léo ; Khoobarry, Kevinee ; Girardet, François ; Burlet, Philippe ; Armand, Anne-Sophie ; Chanoine, Christophe ; Bureau, Jean-François ; Charbonnier, Frédéric ; Lefebvre, Suzie
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2018-02, Vol.8 (1), p.2075-17, Article 2075
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Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy
Author / Creator:Lee, Andrew J-H ; Awano, Tomoyuki ; Park, Gyu-Hwan ; Monani, Umrao R
Publisher:United States: Public Library of Science
Journal title:PloS one, 2012-09, Vol.7 (9), p.e46353-e46353
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Enhanced expression of the central survival of motor neuron (SMN) protein during the pathogenesis of osteoarthritis
Author / Creator:Cucchiarini, Magali ; Madry, Henning ; Terwilliger, Ernest F.
Publisher:England: John Wiley & Sons, Inc
Journal title:Journal of cellular and molecular medicine, 2014-01, Vol.18 (1), p.115-124
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Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

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Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study

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Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs

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SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical Trials

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Survival Motor Neuron (SMN) protein is required for normal mouse liver development

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Analysis of SMN protein in umbilical cord blood and postnatal peripheral blood of neonates with SMA: a rationale for prompt treatment initiation to prevent SMA development

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The phospho-landscape of the survival of motoneuron protein (SMN) protein: relevance for spinal muscular atrophy (SMA)

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LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

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SMN protein promotes membrane compartmentalization of ribosomal protein S6 transcript in human fibroblasts

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Depletion of SMN protein in mesenchymal progenitors impairs the development of bone and neuromuscular junction in spinal muscular atrophy

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Depletion of SMN protein in mesenchymal progenitors impairs the development of bone and neuromuscular junction in spinal muscular atrophy

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A Comparative Study of SMN Protein and mRNA in Blood and Fibroblasts in Patients with Spinal Muscular Atrophy and Healthy Controls

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Recombinant SMN protein synergizes with spinal muscular atrophy therapy to counteract pathological motor neuron phenotypes

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Stability and Oligomerization of Mutated SMN Protein Determine Clinical Severity of Spinal Muscular Atrophy

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Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug‐like compounds

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Calpain Inhibition Increases SMN Protein in Spinal Cord Motoneurons and Ameliorates the Spinal Muscular Atrophy Phenotype in Mice

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Different Stability and Proteasome-Mediated Degradation Rate of SMN Protein Isoforms

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Small-molecule flunarizine increases SMN protein in nuclear Cajal bodies and motor function in a mouse model of spinal muscular atrophy

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Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy

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Enhanced expression of the central survival of motor neuron (SMN) protein during the pathogenesis of osteoarthritis

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