2. HDO Mechanism of Action(1)

2. HDO Mechanism of Action(1)

(RNse H-dependent antisense effect)

Step 1
The HDO carrier strand (RNA) incorporated into the cell is cleaved by RNase H (enzyme).

Step 2
The antisense strand (DNA) remaining after cleavage binds to the target mRNA.

Step 3
When the antisense strand (DNA) binds to mRNA, it becomes DNA / RNA structure again, and RNase H cleaves the target mRNA.

Step 4
As mRNA is cleaved, mRNA expression is suppresses, and drug efficacy is demonstrated.

※RNase H has the property of cleaving RNA among double strands composed of DNA / RNA.

※HDO can also respond to RNaseH-indeoendent antisense effects (e.g., exon skipping)