Asteroid homolog 1 (ASTE1)

Asteroid homolog 1 (ASTE1), commonly referred to in clinical and oncological research as HT001, is a human protein-coding gene that serves as a high-value target for immunotherapy in microsatellite instability-high (MSI-H) cancers. Biologically, ASTE1 functions as a structure-specific DNA endonuclease that facilitates genomic stability by participating in DNA double-strand break repair through both homologous recombination and non-homologous end-joining (NHEJ) pathways. In the context of diseases such as Lynch syndrome and sporadic MSI-H colorectal, gastric, and endometrial cancers, the ASTE1 gene is highly susceptible to translational frameshift mutations, particularly at an A11 mononucleotide repeat sequence. These mutations occur in over 80% of MSI-H cases and result in the expression of unique, highly immunogenic frameshift peptides (FSPs) that are not found in normal tissues. Because these neopeptides are shared across different cancer types and patients, HT001 has become a cornerstone of multi-antigen therapeutic cancer vaccines, such as the Micoryx vaccine. These treatments are designed to prime and activate the patient's immune system to specifically recognize and eliminate tumor cells harboring these specific genetic mutations.

Other names

HT001ASTE1Asteroid-1HT001 neopeptideFSP HT001

Stimulation of a target-specific immune response, including the activation of cytotoxic T lymphocytes and the production of antibodies, against frameshift peptide (FSP) neoantigens generated by mutations in the ASTE1/HT001 gene in mismatch repair-deficient cells.