Zinc finger and CRISPR-Cas9 gene editing are also used to silence genes. Gene silencing might be used if a gene mutation is causing overproduction of a protein.
Another method to ‘turn off’ a gene is RNA interference (RNAi). Specific genes are prevented from producing protein by prevent messenger RNA (mRNA) from creating the disease-causing proteins.
- To make RNAi into medicine, developers need to deliver delicate molecules of RNA safely to their target organs. They need to shield the RNA from degradation in the bloodstream, prevent it from being filtered out by the kidneys, and let it exit blood vessels and spread through tissues.
- Antisense oligonucleotides (ASOs), are short, synthetic DNA fragments that bind RNA through base pairing and modulate its function to silence the effect of the gene.