di-Ellipticine-RIBOTAC TFA is a RNase recruiting chimera (RIBOTAC) degrader, capable of specifically binding and degrading expanded G4C2 RNA repeat (r(G 4 C 2 ) exp ) . di-Ellipticine-RIBOTAC TFA selectively binds the three-dimensional (3D) structure formed by r(G 4 C 2 ) exp and that recruits an endogenous ribonuclease (RNase) to cleave r(G 4 C 2 ) exp . di-Ellipticine-RIBOTAC TFA selectively degrades the mutant chromosome 9 open reading frame 72 ( C9orf72 ) allele and reduces quantities of toxic dipeptide repeat proteins (DPRs) translated from r(G 4 C 2 ) exp . di-Ellipticine-RIBOTAC TFA significantly improves the pathological phenotype of amyotrophic lateral sclerosis/ frontotemporal dementia (c9ALS/FTD) in cells and mouse models. di-Ellipticine-RIBOTAC TFA can be used for the study of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
Products | Products > Disease Areas > Neurological, Eye or Ear Disease | Products > Disease Areas > Neurodegenerative Disease | Products > Disease Areas > Amyotrophic Lateral Sclerosis | Products > Signaling Pathways > PROTAC | Products > Signaling Pathways > Cell Cycle/DNA Damage | Products > Research Areas > Neurological Disease | Products > Signaling Pathways > RIBOTAC | Products > Signaling Pathways > DNA/RNA Synthesis
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