Cerebrospinal Fluid Defines SOD1 as a Pharmacodynamic Marker for Antisense Oligonucleotide Therapy

BACKGROUND: Therapies designed to decrease SOD1 are currently in clinical trial for patients with superoxide dismutase (SOD1)-linked Familial Amyotrophic Lateral Sclerosis (ALS), OBJECTIVE: To determine whether SOD1 protein in cerebral spinal fluid (CSF) may be a pharmacodynamic marker and whether S...

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Autori principali: Winer, Leah, B.A., Srinivasan, Dushyanth, B.A., Chun, Seung, B.A., Lacomis, David, Jaffa, Matthew, B.A., Fagan, Anne, Holtzman, David M., Wancewicz, Ed, B.S., Bennett, C.F., Bowser, Robert, Cudkowicz, Merit, Miller, Timothy M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2013
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3812918/
https://ncbi.nlm.nih.gov/pubmed/23147550
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaneurol.2013.593
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