Inhaled RNA Might Help Heal Cystic Fibrosis
Science Quickly
Scientific American
4.4 • 1.4K Ratings
🗓️ 8 January 2019
⏱️ 2 minutes
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| 0:00.0 | This is |
| 0:02.0 | is Scientific American 60 Second Science. |
| 0:05.0 | I'm Christopher Intagiyata. |
| 0:07.0 | Your genome is sort of like a library, |
| 0:09.0 | with each gene an instruction manual for making proteins. |
| 0:12.0 | The bad news is you can't return a book |
| 0:14.9 | and take out a new edition, but the technique of gene therapy allows you to revise your |
| 0:19.6 | copy of the book so to speak, giving you the ability, potentially, to make new and different proteins. |
| 0:25.9 | Now scientists have experimented with a new way to make text revisions by inhaling the changes. |
| 0:32.2 | They tested the concept by having mice breathe in genetic material called messenger RNA or MRNA. For the test, the |
| 0:39.5 | MRNA included instructions to manufacture Luciferase, the stuff that lights up in fireflies. |
| 0:45.6 | The researchers packaged the MRNA with a degradable polymer to trick the mice's lung cells |
| 0:50.4 | into accepting the package, and once the cells gobbled it up, they began to glow, |
| 0:55.0 | proving that inhaling MRNA is an effective way to kick-start the production of new and novel proteins, |
| 1:01.0 | like the firefly enzyme. |
| 1:03.8 | The scientists also repeated the experiment in mice whose cells had been genetically |
| 1:07.4 | 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. |
| 1:17.0 | The proof of concept is in the journal Advanced Materials. |
| 1:20.0 | Of course, the point of all this isn't to make mice glow. |
| 1:23.0 | Instead, one idea is to use this technique to help cystic fibrosis patients. |
| 1:27.5 | Because people with CF have a genetic mutation that causes a buildup of sticky mucous in their lungs. Several of the study authors that phase one and two trials to determine if inhaling messenger RNA could provide a genetic fix |
| 1:45.2 | for cystic fibrosis. |
... |
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