科学美国人:Inhaled RNA Might Help Heal Cystic Fibrosis

60秒科学节目(SSS)是科学美国人网站的一套广播栏目,英文名称:Scientific American - 60 Second Science,节目内容以科学报道为主,节目仅一分钟的时间,主要对当今的科学技术新发展作以简明、通俗的介绍,对于科学的发展如何影响人们的生活环境、健康状况及科学技术,提供了大量简明易懂的阐释。

Your genome is sort of like a library—with each gene an instruction manual for making proteins. Bad news is, you can’t return a book and take out a new edition. But the technique of gene therapy allows you to revise your copy of the book, so to speak…giving you the ability, potentially, to make new and different proteins.

Now scientists have experimented with a new way to make text revisions—by inhaling the changes. They tested the concept by having mice breathe in genetic material called messenger RNA, or mRNA. For the test, the mRNA included instructions to manufacture luciferase—the stuff that lights up in fireflies. 

The researchers packaged the mRNA with a degradable polymer, to trick the mice's lung cells into accepting the package. And once the cells gobbled it up, they began to glow—proving that inhaling mRNA is an effective way to kick-start the production of new and novel proteins, like the firefly enzyme. 

The scientists also repeated the experiment in mice whose cells had been genetically engineered to turn permanently red if they received a copy of mRNA—making it possible to count the proportion of cells affected by a dose. The proof of concept is in the journal Advanced Materials. [Asha Kumari Patel et al., Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium]

Of course, the point isn't to make mice glow. Instead, one idea is to use this technique to help cystic fibrosis patients. People with CF have a genetic mutation that causes a buildup of sticky mucus in their lungs. Several of the study authors work with a publicly traded biotech company called Translate Bio, which is conducting phase 1 and 2 trials to determine if inhaling messenger RNA could provide a genetic fix for cystic fibrosis. Regardless of the particular case of CF, the luciferase example shows that inhalation genetic therapy could be an inspiration. 

—Christopher Intagliata