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Table 1 Three druggable gene targets of ALS and five existing drugs identified for drugs repurposing application. Tier 1 contains the objectives of the approved drug and clinical drug, tier 2 contains incorporated proteins closely related to drug targets or with associated drug-like compounds, and tier 3 contains incorporated extracellular proteins and members of key drug-target families [8]. All information on medications is from ChEMBL and DrugBank Online

From: TBK1, a prioritized drug repurposing target for amyotrophic lateral sclerosis: evidence from druggable genome Mendelian randomization and pharmacological verification in vitro

Gene

Target

Tier of druggable genes

Tissue

Drug name

Target ChEMBL ID

Action type

Max clinical phase

Indication

TBK1

TANK-binding kinase 1

Tier 1

Blood

Fostamatinib

CHEMBL2103830

Inhibitor

Approved

Immune thrombocytopenic

purpura

Amlexanox

NA

Inhibitor

Approved

Aphthous ulcers

TNFSF12

Tumor necrosis

factor (ligand) superfamily, member 12

Tier 1

Blood

BIIB-023

CHEMBL2109598

Antibody

2

Lupus nephritis; rheumatoid

arthritis

RG-7212

CHEMBL2109600

Antibody

1

Neoplasms

GPX3

Glutathione peroxidase 3 (plasma)

Tier 1

Blood and brain

Glutathione

CHEMBL1543

Cofactor

3

Cystic fibrosis; reperfusion injury; pancreatic neoplasms; Parkinson disease; breast neoplasms; pain; peripheral nervous system diseases; Persian Gulf Syndrome