α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease.

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作者： Tracy A,Cole ^[1] ; Hien,Zhao ^[1] ; Timothy J,Collier ^[2] ; Ivette,Sandoval ^[2] ; Caryl E,Sortwell ^[2] ; Kathy,Steece-Collier ^[2] ; Brian F,Daley ^[2] ; Alix,Booms ^[2] ; Jack,Lipton ^[2] ; Mackenzie,Welch ^[3] ; Melissa,Berman ^[3] ; Luke,Jandreski ^[3] ; Danielle,Graham ^[3] ; Andreas,Weihofen ^[3] ; Stephanie,Celano ^[2] ; Emily,Schulz ^[2] ; Allyson,Cole-Strauss ^[2] ; Esteban,Luna ^[4] ; Duc,Quach ^[1] ; Apoorva,Mohan ^[1] ; C Frank,Bennett ^[1] ; Eric E,Swayze ^[1] ; Holly B,Kordasiewicz ^[1] ; Kelvin C,Luk ^[4] ; Katrina L,Paumier ^[2]

作者单位： Ionis Pharmaceuticals Inc., Carlsbad, California, USA. ^[1] Michigan State University, Grand Rapids, Michigan, USA. ^[2] Biogen, Cambridge, Massachusetts, USA. ^[3] Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania, USA. ^[4]

关键词 Neuroscience Parkinson disease Therapeutics

主题词动物(Animals);脑(Brain);细胞培养技术(Cell Culture Techniques);脑脊髓液(Cerebrospinal Fluid);疾病模型, 动物(Disease Models, Animal);女(雌)性(Female);人类(Humans);成束猴(Macaca fascicularis);男(雄)性(Male);小鼠(Mice);寡核苷酸类, 反义(Oligonucleotides, Antisense);帕金森病(Parkinson Disease);RNA, 信使(RNA, Messenger);大鼠, Sprague-Dawley(Rats, Sprague-Dawley);α突触核蛋白(alpha-Synuclein);大鼠(Rats)

DOI 10.1172/jci.insight.135633

PMID 33682798

发布时间 2024-02-26

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2021年6卷5期

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α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease.

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α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson's disease.

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