Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC 50 of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15 , and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis.
Products | Products > Disease Areas > Neurological, Eye or Ear Disease | Products > Disease Areas > Neurodegenerative Disease | Products > Disease Areas > Amyotrophic Lateral Sclerosis | Products > Signaling Pathways > Immunology/Inflammation | Products > Research Areas > Neurological Disease | Products > Signaling Pathways > SOD
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