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Oligonucleotide therapeutics are short synthetic strands of genetic code that silence a specific cancer gene.
Oligonucleotide therapeutics are short synthetic nucleic acid strands that base-pair to a target mRNA and induce RNase H cleavage (antisense) or RISC-mediated degradation (siRNA), silencing a gene at the transcript level. In principle any gene is targetable, including undruggable transcription factors. Antisense and siRNA drugs are established outside oncology; in cancer, delivery to tumours is the barrier, since existing chemistries concentrate in the liver. Antibody-oligonucleotide conjugates and lipid nanoparticle delivery are in early trials, with targets including KRAS, STAT3, and MYC. Whether enough drug can reach solid tumours to silence a driver is the open question. The simple version is a synthetic strand of genetic code that switches off one cancer gene, if it can be delivered.
Base-pairing to mRNA induces RNase H cleavage or RISC-mediated degradation.
A drug designed to switch off a protein that helps cancer cells survive chemotherapy. Two large trials found it added nothing.
Imetelstat is a lipid-linked oligonucleotide that binds the RNA template of telomerase, the enzyme cancer cells use to stay immortal, and it is the first telomerase inhibitor approved for any cancer. In lower-risk myelodysplastic syndromes it freed 40% of transfusion-dependent patients from transfusions for at least eight weeks versus 15% on placebo, once growth factors have failed.
Query for this technology: (TITLE:"antisense oligonucleotide" OR ABSTRACT:"antisense oligonucleotide" OR TITLE:"siRNA" OR ABSTRACT:"siRNA") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Oligonucleotide therapeutics, not a curated reading list.
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