Genetic instructions for how to build the huntingtin protein are stored inside the nucleus, in tightly bound chains of molecules called DNA

Courier molecules inside the cells, called messenger RNA, transcribe the information from the DNA and transport it to ribosomes in the cell's cytoplasm where the protein is made

Reduced

protein

production

Source: Ionis Pharmaceuticals

Gene silencing

mRNA

The experimental drug, IONIS-HTTRx, gets inside the cell and sticks to the huntingtin mRNA before it reaches the ribosomes, flagging it for deletion by the cell's RNA recycling machinery—the more mRNA it intercepts, the less mutant huntingtin protein is made

DNA

How gene silencing works

Genetic instructions for how to build the huntingtin protein are stored inside the nucleus, in tightly bound chains of molecules called DNA

Courier molecules inside the cells, called messenger RNA, transcribe the information from the DNA and transport it to ribosomes in the cell's cytoplasm where the protein is made

Reduced

protein

production

Source: Ionis Pharmaceuticals

Gene silencing

mRNA

DNA

The experimental drug, IONIS-HTTRx, gets inside the cell and sticks to the huntingtin mRNA before it reaches the ribosomes, flagging it for deletion by the cell's RNA recycling machinery—the more mRNA it intercepts, the less mutant huntingtin protein is made

How gene silencing works

Reduced

protein

production

Gene silencing

Genetic instructions for how to build the huntingtin protein are stored inside the nucleus, in tightly bound chains of molecules called DNA

Source: Ionis Pharmaceuticals

mRNA

DNA

Courier molecules inside the cells, called messenger RNA, transcribe the information from the DNA and transport it to ribosomes in the cell's cytoplasm where the protein is made

The experimental drug, IONIS-HTTRx, gets inside the cell and sticks to the huntingtin mRNA before it reaches the ribosomes, flagging it for deletion by the cell's RNA recycling machinery—the more mRNA it intercepts, the less mutant huntingtin protein is made

How gene silencing works

Reduced

protein

production

Source: Ionis Pharmaceuticals

Gene silencing

mRNA

DNA

Courier molecules inside the cells, called messenger RNA, transcribe the information from the DNA and transport it to ribosomes in the cell's cytoplasm where the protein is made

The experimental drug, IONIS-HTTRx, gets inside the cell and sticks to the huntingtin mRNA before it reaches the ribosomes, flagging it for deletion by the cell's RNA recycling machinery—the more mRNA it intercepts, the less mutant huntingtin protein is made

Genetic instructions for how to build the huntingtin protein are stored inside the nucleus, in tightly bound chains of molecules called DNA

How gene silencing works