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