Drug intelligence / Profile preview

lenti-miR449a

Development stage
Preclinical
Lead developer
AptamiR Therapeutics
Modality
Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral, Intravenous
01

Overview

lenti-miR449a is a lentiviral vector-based gene therapy designed to overexpress microRNA-449a (miR-449a). In the context of bladder cancer research, it is used to down-regulate the androgen receptor (AR) signaling pathway. miR-449a acts as a tumor suppressor by targeting the expression of AR and its downstream effector, prostate-specific antigen (PSA), as well as inducing G0/G1 cell cycle arrest through the inhibition of HDAC1 or Rb phosphorylation. By increasing miR-449a levels, lenti-miR449a inhibits the proliferation and viability of AR-positive bladder cancer cells.

Other names
lentivirus-miR-449alentivirus-miR449alentivirus-miR 449amiR-449a lentivirusmiR449a lentivirusmiR 449a lentivirus
02

Targets

Cyclin-dependent kinase 6 mRNA 3' untranslated region (CDK6 mRNA 3' UTR)LEF1CCND1 (Cyclin D1)TSEN15 (tRNA-splicing endonuclease subunit 15)Cell division cycle 25A mRNA 3' untranslated regionPlakophilin 4 mRNA 3' untranslated region (PKP4 mRNA 3' UTR)Acyl-CoA synthetase long-chain family member 4 mRNA 3' UTR

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