作者
Leah Winer, Dushyanth Srinivasan, Seung Chun, David Lacomis, Matthew Jaffa, Anne Fagan, David M Holtzman, Ed Wancewicz, C Frank Bennett, Robert Bowser, Merit Cudkowicz, Timothy M Miller
发表日期
2013/2/1
期刊
JAMA neurology
卷号
70
期号
2
页码范围
201-207
出版商
American Medical Association
简介
Background
Therapies designed to decrease the level of SOD1 are currently in a clinical trial for patients with superoxide dismutase (SOD1)–linked familial amyotrophic lateral sclerosis (ALS).
Objective
To determine whether the SOD1 protein in cerebral spinal fluid (CSF) may be a pharmacodynamic marker for antisense oligonucleotide therapy and a disease marker for ALS.
Design
Antisense oligonucleotides targeting human SOD1 were administered to rats expressing SOD1G93A. The human SOD1 protein levels were measured in the rats' brain and CSF samples. In human CSF samples, the following proteins were measured: SOD1, tau, phosphorylated tau, VILIP-1, and YKL-40.
Participants
Ninety-three participants with ALS, 88 healthy controls, and 89 controls with a neurological disease (55 with dementia of the Alzheimer type, 19 with multiple sclerosis, and 15 with peripheral neuropathy).
Results
Antisense …
学术搜索中的文章
L Winer, D Srinivasan, S Chun, D Lacomis, M Jaffa… - JAMA neurology, 2013