ADAR1 shRNA

ADAR1 shRNA is a gene-silencing therapeutic candidate designed to knock down the expression of Adenosine Deaminase Acting on RNA 1 (ADAR1). ADAR1 is an enzyme that catalyzes the deamination of adenosine to inosine (A-to-I) in double-stranded RNA, a process that is often deregulated in various cancers. In multiple myeloma (MM), ADAR1 is frequently overexpressed due to chromosome 1q21 amplification and inflammatory cytokine stimulation, leading to aberrant RNA editing of targets like GLI1. This editing promotes malignant self-renewal and therapeutic resistance. By utilizing short hairpin RNA (shRNA) delivered via lentiviral vectors, researchers can reduce ADAR1 levels, thereby inhibiting the Hedgehog pathway activator GLI1 and sensitizing MM cells to treatments like lenalidomide. Preclinical studies in humanized mouse models have demonstrated that ADAR1 knockdown reduces the regeneration of high-risk MM cells and modulates extracellular and immune response genes.

Other names

ADAR1 short hairpin RNAADAR-1 short hairpin RNAADAR 1 short hairpin RNAshRNA targeting ADAR1