Research into RNA interference has come a long way in the last decade and has the potential for a wide array of new therapies, according to the Department of Molecular Biology’s Daniela Castanotto, Ph.D., and John J. Rossi, Ph.D. The pair recently reviewed the state of the field in the January issue of Nature.
 John Rossi and Daniela Castanotto (Photo by Darrin S. Joy) |
For decades after its discovery, researchers viewed RNA — or ribonucleic acid — as little more than a “middle man” molecule, simply helping the cell translate the genetic code from DNA into proteins.
Now researchers know that different forms of RNA can do much more, including actually interfering with translation of the genetic code. This discovery made it clear that RNA, in its different forms, helps regulate genes.
In Nature, Castanotto, associate research scientist, and Rossi, Lidow Family Research Chair in the Department of Molecular Biology, gave a brief history of how “the RNA world” has gained attention in the scientific community. They detailed how RNA interference works, noting that just a decade from its discovery, the phenomenon already is being used therapeutically in human clinical trials.
Over the past 15 to 20 years, researchers have been studying how to use forms of RNA to control cellular processes and fight disease. Rossi and his City of Hope colleagues were among the first to target a disease this way when they used ribozymes — sometimes called “molecular scissors” — to block HIV in patients with AIDSrelated lymphoma. Since then, they have turned their sites to focus on RNA interference to fight HIV and other diseases. Their work resulted in a clinical trial now under way studying RNA interference as a treatment for HIV — the first-ever RNA-interference-based gene therapy trial for treatment of HIV infection.
In their review, Rossi and Castanotto described advances in the field as well as technical challenges, safety issues, the field’s rapidly expanding economic impact and the myriad diseases RNA interference could treat.
According to the review, researchers around the world already are testing RNA interference to treat Parkinson’s disease, type 2 diabetes, rheumatoid arthritis and cancer among others, with the field poised to expand dramatically in the coming years.
“Given the way that [RNA interference] has transformed basic research and the unprecedented speed with which it has reached the clinic,” they wrote, “the coming years promise to be exciting.”
