SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy
L Winer, D Srinivasan, S Chun, D Lacomis… - JAMA …, 2013 - jamanetwork.com
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 SOD1 G93A. The human SOD1 protein levels were …
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 SOD1 G93A. The human SOD1 protein levels were …