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Basic medical and genetic research in the last years has shown that tumors and many other as yet 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.
Employing antisense molecules to inhibit the synthesis of such pathogenic proteins is an innovative therapeutic approach. These molecules consist of small RNA or DNA segments (oligonucleotides), which attach to the messenger RNA, so that they cannot be read on the ribosomes (translation).
In this way, 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.
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