RNAi

Question 1

What is shRNA?

Answer 1

Human made short hairpin RNA that exploits the endogenous miRNA machinery to silence the expression of certain genes

Question 2

How does RNAi work?

Answer 2

1. A plasmid encoding the shRNA is designed and introduced into the cell
2. The shRNA gets processed by the endogenous machinery and generates siRNA
3. The siRNA binds to the target with perfect complementarity and the mRNA gets cleaved and degraded

Question 3

Is RNAi an endogenous mechanism of gene regulation?

Answer 3

Nope

Question 4

Why is RNAi a potential therapy for disease treatment?

Answer 4

It can target and knock down disease specific gene expression

Question 5

What are 3 challenges with RNAi based therapies?

Answer 5

1. Delivery - how to get the drug to the place it needs to be
2. Immune response - mammals will mount an immune response against dsRNA
3. Target and off-target genes - how to we get it to target only what we want and nothing else

Question 6

What two organs are the best candidates for RNAi based therapies? Why?

Answer 6

Eye and liver diseases. They easy to deliver the shRNA to

Question 7

Why is RNAi promising in agriculture?

Answer 7

Environmentally friendly alternative to pesticides, since RNA doesn’t persist in the environment