Q&A

What is antisense technology?

What is antisense technology?

Antisense DNA technology is a method to inhibit or downregulate the production of a target protein by using antisense DNA or RNA molecules. An antisense sequence is a DNA or RNA that is perfectly complementary to the target nucleotide sequence present in the cell.

What are two types of RNAi?

Two types of small ribonucleic acid (RNA) molecules – microRNA (miRNA) and small interfering RNA (siRNA) – are central to RNA interference.

What is antisense gene therapy?

Antisense therapy is an approach to fighting diseases using DNA-like molecules (AOs). After initially observing antisense-mediated RNA regulation in nature, investigations using model systems to test the feasibility of using synthetic AOs to reduce levels of specific mRNA transcripts quickly followed.

How do antisense oligonucleotides work?

Antisense oligonucleotides are synthetic fragments of DNA that can bind to mRNAs, causing them to be cut into pieces. An important property of mRNAs is that the precise code they contain is specific to each protein.

Is antisense oligonucleotide a gene therapy?

Genetic therapies such as antisense oligonucleotide (ASOs) and RNA interference (RNAi) do exactly this. Through canonical Watson-Crick base pairing (figure 1A), these drugs bind individual RNAs to modulate gene expression and thus protein availability.

What’s the difference between antisense and RNAi therapy?

Like antisense therapy, RNAi is a gene silencing technique that inhibits the actions of genes by interfering with the translation of proteins. However, antisense technology destroys target mRNA by recruiting the enzyme RNase H, while RNAi recruits a different RNase enzyme known as dicer.

What’s the difference between antisense and micro RNA?

micro RNA In The Mix. Antisense and RNAi are both synthesized in the lab and delivered to patients to decrease the expression of a disease-associated protein. MicroRNA (miR), on the other hand, is a type of dsRNA made by cells to regulate gene expression.

Are there any RNAi therapies on the market?

Alnylam Pharmaceuticals is preparing to launch the first siRNA drug this year, targeting an inherited disease called ATTR amyloidosis, and numerous other companies are racing to develop RNAi therapies. The second kind of RNA therapy focuses on replacing mRNA.

What are the different types of RNA therapy?

(In comparison, we still don’t know how many drugs — including Tylenol — actually work.) There are two main types of RNA therapy, antisense – which degrades dysfunctional or harmful proteins in the cell – and mRNA, which produces functional proteins. Currently, antisense therapy is more common, though more complicated.