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Prior to the discovery of RNA interference (RNAi), in vitro
knock-out of genes was technically troublesome and time-consuming
work. Mostly, antisense molecules or ribozymes were used for
it, but it often caused side effects and the results were not
easy to interpret. RNAi is a mechanism of gene silencing at
the mRNA level and offers a quick and effective approach for
shutting off specific genes.
Especially synthetic small interfering RNA (siRNA) offers
researchers an easy and consistent tool to manipulate genes
in vitro. siRNA as a duplex is typically made of 19 bases
with symmetric 2 nucleotide 3กฏ overhangs and can target any
genes. Nowadays, the popularity of RNAi has increased rapidly
as a tool for functional genomics and target validation, replacing
the expensive transgenic animals for gene silencing experiments.
RNAi also has the potential to be used as a powerful therapeutic
with high specificity. Two RNA-based drugs are already submitted
for Phase I clinical study in the USA to cure age-related
macular degeneration (AMD) and more are expected to follow
this year.
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