Sirna inhibiting expression of amyloid precursor protein (app) gene, drug, and use
AssigneeBeBetter Med Inc.
InventorsFushun FAN, Xinjian LIU, Yingying LIN, Tiancheng FU, Changgeng QIAN
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AssigneeBeBetter Med Inc.
InventorsFushun FAN, Xinjian LIU, Yingying LIN, Tiancheng FU, Changgeng QIAN
Estimating the term…
The present invention provides siRNA, peptide oligonucleotide drugs, and their applications for suppressing the expression of the amyloid precursor protein (APP) gene in human cells. The siRNA exhibits potent activity in inhibiting APP expression. Through appropriate modifications, its ability to silence the target is enhanced while reducing off-target activity. The described siRNA and its conjugates hold promise for clinical application in the prevention and treatment of diseases associated with the APP target, including cerebral amyloid angiopathy (CAA), early-onset familial Alzheimer's disease (EOFAD), or Alzheimer's disease (AD).
An siRNA or a pharmaceutically acceptable salt thereof for suppressing the expression of the amyloid precursor protein (APP) gene, wherein the siRNA comprises a sense strand and a corresponding antisense strand, and wherein the siRNA is selected from any of the following pairs of sequences, or sequences differing from its sense strand or antisense strand by no more than one nucleotide:
Dependencies are inferred from the claim text.