1. HDO Structure
HDO is composed as follows. Antisense strand (DNA, Gapmer, PMO etc.): Has a medicinal effectCarrier strand (RNA): Transport active strand (medicina
2. HDO Mechanism of Action（1）
(RNase H-dependent antisense effect)Step 1The HDO carrier strand (RNA) incorporated into the cell is cleaved by RNase H (enzyme).Step 2The antisen
3. HDO Mechanism of Action（2）
(Splicing Control)Step 1HDO enters the cell through some special ligand into the cells.Step 2The carrier stand cleaved by RNase.Step 3The antise
4. Strengths of HDO technology
4. Strengths of HDO technology
HDO has the following strengths:By linking the ligand to the carrier strand, it is possible to selectively deliver the antisense strand to the diseas
5. Biological activity of HDO
Comparison of knockdown activity of HDO and ASO in mouse liver
6. Advantages of HDO: Nuclear translocation
6. Advantages of HDO: Nuclear translocation
Right: Strong and distinct nuclear signal of ASO was observed in most HDO-transfected cells.Left: Diffuse or partially scattered weak signal througho
