Gene-specific therapies and their mechanisms
The new category of oligonucleotide gene-specific medicines is characterised by a short chemical sequence of modified DNA that acts on a specific pathological target gene.
Anti-gene
An anti-gene is an oligonucletoide able to inhibit the transcripstion of a target gene ino mRNA
Target gene
(two copies per cell)
Mechanism
Mechanism The anti-gene oligonucletoide works by forming a stable duplex with the complementary DNA sequence, binding to the open DNA structure of an active gene, thereby blocking transcription
mRNA
(Thousands of copies)
Applications
MyGeneraTM technology is applicable to any disease where selective silencing of a target gene will have therapeutic effect eg. oncology, dominant genetic diseases, viral/bacterial infections.
Protein
(Thousands of copies)