RNA is the messenger molecule that conveys information from the genome and allows
the production of biomolecules required for life in a responsive and regulated way. Most
genes are able to produce multiple mRNA products in response to different internal
or external environmental signals, in different tissues and organs, and at ...
RNA is the messenger molecule that conveys information from the genome and allows
the production of biomolecules required for life in a responsive and regulated way. Most
genes are able to produce multiple mRNA products in response to different internal
or external environmental signals, in different tissues and organs, and at specific times
in development or later life. This fine tuning of gene expression is dependent on the
coordinated effects of a large and intricate set of regulatory machinery, which together
orchestrate the genomic output at each locus and ensure that each gene is expressed
at the right amount, at the right time and in the correct location. This complexity of
control, and the requirement for both sequence elements and the entities that bind them,
results in multiple points at which errors may occur. Errors of RNA biology are common
and found in association with both rare, single gene disorders, but also more common,
chronic diseases. Fortunately, complexity also brings opportunity. The existence of many
regulatory steps also offers multiple levels of potential therapeutic intervention which
can be exploited. In this review, I will outline the specific points at which coding RNAs
may be regulated, indicate potential means of intervention at each stage, and outline
with examples some of the progress that has been made in this area. Finally, I will
outline some of the remaining challenges with the delivery of RNA-based therapeutics
but indicate why there are reasons for optimism.
