Basic medical and genetic research in recent years has shown that tumors and many other incurable diseases are caused by defective regulation of certain genes. This manifests as excessive or faulty synthesis of proteins that can trigger tumor diseases and promote their progression.
The use of antisense molecules to inhibit the synthesis of such pathogenic proteins represents an innovative therapeutic approach. Antisense compounds are biological molecules consisting of small ribonucleic acid (RNA) or deoxyribonucleic acid (DNA) segments (oligonucleotides), which attach to the messenger RNA, resulting in the inhibition of protein synthesis (translation).
Accordingly, these drugs enable a causal therapy of certain diseases, without altering the patient’s genome. Antisense molecules are highly specific for a certain messenger RNA and therewith for certain proteins.
On average, less than 1% of the messenger RNA is a suitable target for antisense oligonucleotides. Within this 1%, only few sequence segments meet all the criteria to make a suitable antisense molecule successful as a therapeutic medication.