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SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
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SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
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Journal Article
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
2020
Overview
Two patients with amyotrophic lateral sclerosis caused by
SOD1
mutations received an intrathecal infusion of adeno-associated virus containing microRNA targeting
SOD1
. SOD1 protein levels did not change in the patients’ cerebrospinal fluid, but one patient had reduced spinal cord SOD1 expression.
Publisher
Massachusetts Medical Society
Subject
/ Amyotrophic Lateral Sclerosis - cerebrospinal fluid
/ Amyotrophic Lateral Sclerosis - genetics
/ Amyotrophic Lateral Sclerosis - therapy
/ Autopsy
/ Humans
/ Hypoxia
/ Male
/ miRNA
/ Mutation
/ Patients
/ Proteins
/ Superoxide Dismutase-1 - analysis
/ Superoxide Dismutase-1 - cerebrospinal fluid
