BioQuakes

AP Biology class blog for discussing current research in Biology

Tag: cystic fibrosis

Inhale RNA, Exhale Your Worries

The focus of a recent study is inhalation genetic therapy to give patients with Cystic Fibrosis relief when they breathe. A defective gene in people with Cystic Fibrosis causes a mucus build-up in specific organs. The respiratory complications due to mucus build-up in the lungs are which infections, clogged airways, inflammation, and respiratory failure.

Recently, scientists developed a study that involves mice inhaling messenger RNA. The messenger ribonucleic acid is genetically manipulated so that it contains an oxidative enzyme called “luciferase”, which is known for causing bioluminescence. Scientists manipulated the mRNA by “packaging” or combining the enzyme with a polymer that would be inhaled into the lungs of the mice. The inhaled polymer would then travel through the respiratory system and be taken in by the lungs, where it would eventually be broken down by cells within the lungs. Scientists were able to distinguish if the experiment was successful as the light from the luciferase combined with the mRNA could be detected from within a lung cell.

Another experiment was conducted with similar circumstances in that it tested genetically modified mice cells that glowed red from the cell’s reception of mRNA. This offered the scientists the opportunity to test a range of mRNA-polymer dosages to quantify or count the resulting “red” mice cells.

As we continue this road down modern medicine, mRNA can be evolutionary for patients with Cystic Fibrosis because the messenger RNA can recreate functional copies of itself to produce CFTR protein (cystic fibrosis transmembrane conductance regulator protein), which is the protein that codes and determines the functionality of the CFTR gene. Could mRNA polymers possibly be a treatment for milder respiratory issues like asthma? This experiment might just be a breakthrough in the world of medicine, as strands of ribonucleic acid could be the answer to ending compromising respiratory complications.

*Sing in Rihanna’s voice* Breath out, Breathe in (mRNA)… American Oxygen!

Researches at the Massachusetts Institute of Technology (MIT) have designed a potentially groundbreaking tool for helping treat lung disease. Their design? One might find the answer rather surprising: inhalable mRNA.

What is mRNA?

Also known as messenger ribonucleic acid, mRNA is a subunit of RNA, and is responsible for carrying the genetic information copied from DNA in the form of a code. More specifically, mRNA is synthesized during transcription. As explained in the article, mRNA, “encodes genetic instructions that stimulate cells to produce specific proteins.” Click here to learn more about mRNA.

The Benefits:

Inhalable mRNA? Yes, you read that correctly. Essentially, patients would inhale the mRNA in an aerosol form. By doing such, the mRNA would come into direct contact with the patient’s lung’s cells, which would then trigger the production of “therapeutic” proteins. As stated in the article, such mRNA molecules, “[turn] the patients’ own cells into drug factories.” If done successfully, mRNA has the potential to treat a myriad of lung-related illnesses, cystic fibrosis among them. Daniel Anderson, an associate professor in MIT’s Department of Chemical Engineering, expresses confidence regarding the findings, stating, “We think the ability to deliver mRNA via inhalation could allow us to treat a range of different disease of the lung.”

Obstacles:

Presently, scientists face the challenge of targeting cells with the mRNA aerosol molecules by using methods which are both safe and efficient. Additionally, scientists are tasked with the challenge of transporting these mRNA molecules in protective carriers, as the body’s natural reaction is to break mRNA down.

The Experiment:

In order to determine the impact of inhalable mRNA, Dr. Daniel Anderson has successfully manipulated a mice’s lung cells to produce a target protein. Dr. Anderson and his lab have begun designing materials which can transport mRNA to organs such as the liver. In particular, he and his lab utilized polyethylenimine (PEI), as it doesn’t break down easily. However, this very aspect of the polymer has the potential to cause side effects. In an effort to avoid these unwanted symptoms, the team moved on to a biodegradable material called “hyperbranched poly”. To test this material, the scientists converted the material into a droplet form, using a nebulizer to deliver the inhalable mist to a group of mice. Twenty four hours later, the team found that the mice were indeed producing the sought-after bioluminescent protein. Moreover, with the decrease in mRNA dosage came the decrease in protein production.

Pictured above is polyethylenimine (PEI), the initial polymer used in Dr. Anderson’s experiment.

The Future of Inhalable mRNA:

Such developments, such as those performed by Dr. Anderson and his team, increase the potential reality of testing on patients. To read the full findings of the aforementioned experiment, click here.

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