The RNA Miracle Unfolds

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The RNA Miracle Unfolds

RNA interference (RNAi) was first observed in the 1990s, and by 2006 the researchers who identified the phenomenon received the Nobel Prize.  We’ve discussed RNAi in previous issues, so we’ll refer you to those discussions, and quickly remind you that it is a biological process that organisms use to keep genes from being expressed by binding tiny pieces of RNA to the complementary sequences of genes. 

Most importantly, RNAi can help the body to resist viral infection by switching off the genes the virus needs to replicate.  According to MIT Technology Review, U.S. researchers are currently conducting more than 150 clinical trials that use RNA to treat such diseases and medical conditions as cancer, infection, hormonal imbalances, and brain diseases such as Huntington’s.1

An examination of a few of those new studies provides a glimpse into how this line of research is creating effective solutions to many of the world’s most urgent challenges.

Let’s begin with a promising approach to providing protection against HIV.  A team led by scientists from the Perelman School of Medicine at the University of Pennsylvania has demonstrated a new way to deliver safer and more cost-effective therapeutic antibodies to treat disease.

According to the researchers, five of the ten best-selling drugs in the United States are based on antibodies, but the downside is that antibodies are large, complex proteins that can be expensive to manufacture.  The new technique involves the injection of messenger RNAs (mRNAs), which are the “blueprint” molecules that cells use to manufacture proteins.

The mRNA molecules are absorbed by cells in the body, which then become factories for making the therapeutic proteins—in this case, antibody proteins—encoded by the mRNAs.

These mRNAs are modified so they can easily enter cells and not activate inflammatory molecules that lead to adverse events.  Researchers have previously shown that they can use this method to make hormones and other proteins in lab animals. 

In the new study, reported in Nature Communications, the team demonstrated that in mice the mRNA method can be used to make enough antibody proteins to deliver vaccine-like protection against HIV in a single dose.2

According to senior author Drew Weissman, MD, PhD, a professor of Infectious Diseases, “We showed that you can give 1/40th the dose of mRNA compared to the antibody protein itself, and completely protect mice from HIV infection when they are exposed to the virus...

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