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from the Rochester Democrat and Chronicle by Stephen Dewhurst –
The ability to sequence the human genome has transformed the way we approach science and medicine. But understanding our DNA is really only the first step in the Genomics revolution.
The same DNA is present in each and every cell of our bodies – yet each of those cells is strikingly different, and optimized to fulfill a different function. That’s because each of those cells produces a completely different set of RNA molecules – its so-called “transcriptome.”
RNA is chemically similar to DNA, and is copied from DNA, often to provide a working copy or blueprint. Indeed, the central dogma of molecular biology holds that DNA codes for messenger RNA (mRNA), and that mRNA is then used to produce the proteins which make up our cells and our bodies. However, the Human Genome Project revealed that over 90% of our DNA doesn’t encode any protein at all – but much of it does encode RNA.
It turns out that the RNAs produced in our cells are much more complex and important than was originally anticipated. They include not only RNAs involved in protein synthesis (such as mRNA), but also RNAs that regulate gene expression, DNA replication and the metabolism of other RNAs. That’s without mentioning parasitic RNAs that seek simply to propagate themselves.
These RNAs vary enormously both in function and in size – from tiny short interfering RNAs and microRNAs, to long non-coding RNAs that control gene expression. What they have in common is the ability to dramatically modify and diversify the proteins produced by our DNA. As a result, RNAs are a key determinant of many biological processes, including development, immunity and even cancer.
RNA also represents a tantalizing drug target. Right now, the drugs we use to treat diseases are almost all directed against proteins. However, the slowing pace and spiraling cost of new drug discovery makes it clear that new approaches are needed. In this sense, the RNA world presents a completely new universe of potentially druggable targets with enormous therapeutic potential, and is only just beginning to be tapped by companies such as Alnylam.
This is a field in which the UR has a clear lead, and the potential to make a distinctive contribution. For example, Dr. Charles Thornton has an RNA target in clinical trial for myotonic dystrophy, in partnership with Ionis Pharmaceuticals, while Drs. Lynne Maquat and Yi-Tao Yu are working to develop agents that regulate RNA metabolism in diverse disease states.
Ultimately, the next wave in genomics – Genomics 2.0 – will be to understand RNA Biology, and to use that knowledge to develop new precision medicines that target disease pathways, with greater effectiveness and reduced side effects.
Source – Rochester Democrat and Chronicle
