antisense oligonucleotide targeting HERV-K Env

This antisense oligonucleotide (ASO) is designed to target the envelope (Env) protein of the Human Endogenous Retrovirus-K (HERV-K), specifically the HML-2 subtype. HERV-K Env is transcriptionally active in glioblastoma (GBM) and other cancers, where it functions as a critical component of the stem cell niche. The ASO works by knocking down HERV-K Env expression, thereby disrupting a novel oncogenic signaling axis involving CD98hc-mediated activation of EGFR. Preclinical studies conducted by the University of Miami and the National Institute of Neurological Disorders and Stroke (NINDS) have shown that this ASO reduces GBM cell viability and spheroid formation in vitro and prolongs survival in orthotopic murine models. The therapy specifically targets tumor cells without affecting normal human astrocytes, suggesting a high degree of precision for treating treatment-resistant tumor stem cells.

Other names

HERV-K Env ASOantisense oligonucleotide targeting HERV-K envelope