Researchers plumbing mysterious RNAs find a possible treatment

WWhen hundreds of scientists from around the world finally pieced together a draft of the first human genome in 2003, perhaps the biggest surprise was how little of it was devoted to producing proteins. About 98% of the genes in our chromosomes seemed to do nothing, earning the unflattering nickname “junk DNA.” But with better tools developed over the past 20 years, scientists began to discover that all that junk actually produces a diverse menagerie of RNA species that are transcribed and released to float through the cell.

Figuring out exactly what everyone is doing is the mission of an increasing number of researchers, including a team from the MD Anderson Cancer Center in Texas. Years ago, they wanted to determine the functions of one RNA species in particular — large ones, larger than 200 bases, known as long noncoding RNAs or lncRNAs. Now they’ve linked one of these RNA molecules to a discovery they believe could help develop new treatments for one of the most common genetic disorders, phenylketonuria.