SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy.

ABSTRACT:

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

SUBMITTER: Winer L 

PROVIDER: S-EPMC3812918 | biostudies-literature | 2013 Feb

REPOSITORIES: biostudies-literature