Multi-omics profiling of CSF from spinal muscular atrophy type 3 patients after nusinersen treatment: a 2-year follow-up multicenter retrospective study.

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作者： Irene,Faravelli ^[1] ; Delia,Gagliardi ^[2] ; Elena,Abati ^[3] ; Megi,Meneri ^[2] ; Jessica,Ongaro ^[3] ; Francesca,Magri ^[2] ; Valeria,Parente ^[3] ; Lucia,Petrozzi ^[3] ; Giulia,Ricci ^[3] ; Fiorenza,Farè ^[3] ; Giulia,Garrone ^[4] ; Manuela,Fontana ^[4] ; Donatella,Caruso ^[5] ; Gabriele,Siciliano ^[5] ; Giacomo Pietro,Comi ^[5] ; Alessandra,Govoni ^[5] ; Stefania,Corti ^[6] ; Linda,Ottoboni ^[4]

作者单位： Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy. irene.faravelli@unimi.it. ^[1] Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy. ^[2] Neurology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. ^[3] Department of Clinical and Experimental Medicine, Neurological Clinics, University of Pisa, Pisa, Italy. ^[4] Unitech OMICs, University of Milan, Milan, Italy. ^[5] Department of Pharmacological and Biomolecular Sciences, Università degli Studi di Milano, Milan, Italy. ^[6] Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy. stefania.corti@unimi.it. ^[7] Neurology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. stefania.corti@unimi.it. ^[8] Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Milan, Italy. linda.ottoboni@unimi.it. ^[9]

关键词 Antisense oligonucleotides Metabolomic Proteomic Spinal muscular atrophy

主题词人类(Humans);回顾性研究(Retrospective Studies);随访研究(Follow-Up Studies);蛋白质组(Proteome);肌萎缩, 脊髓性(Muscular Atrophy, Spinal)

DOI 10.1007/s00018-023-04885-7

PMID 37543540

发布时间 2023-08-14

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