Cutting-Edge Gene Editing Nanoparticles Developed By MIT To Fight Lung Diseases

by Liam O'Connor
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Scientists want to use special particles that can send RNA into the body to help people with diseases like cystic fibrosis and other illnesses of the lungs.

Researchers from MIT and the University of Massachusetts Medical School have created nanoparticles that bring proteins to the lungs. These protein-bringing particles could help fight diseases like cystic fibrosis, which is a disease that hurts your lungs. In a study on mice, these particles successfully carried CRISPR/Cas9 gene-editing components which would help replace genes that cause diseases. The researchers are now working on making it safe so people with lung diseases can breathe in the nanoparticles. They plan to test it in mice with cystic fibrosis and other lung issues soon.

Scientists from Massachusetts Institute of Technology (MIT) and University Of Massachusetts Medical School have created a special type of tiny particle, called ‘nanoparticles’. These particles can be breathed into the lungs and help to deliver proteins useful for treating illnesses such as cystic fibrosis. With more work on these nanoparticles, researchers believe they could be used to treat diseases in the lungs.

Researchers at MIT have discovered a way to efficiently deliver RNA to mouse lungs, which could be used to treat and fix genetic diseases like Cystic Fibrosis. Professor Daniel Anderson, who works at both MIT’s Chemical Engineering Department and the Koch Institute for Integrative Cancer Research and IMES commented on the discovery.

Scientists used tiny particles to deliver a specific genetic medicine in a study involving mice. This could allow us to design treatments that could get rid of genes that cause diseases and replace them with healthy ones.

Three people, Anderson, Professor Robert Langer from MIT and Professor Wen Xue from UMass Medical School RNA Therapeutics Institute, wrote the study that was published on March 30 2023 in Nature Biotechnology. The lead authors are Bowen Li who used to be a postdoc at MIT but is now an assistant professor at University of Toronto; Rajith Singh Manan who is still a postdoc with MIT and Shun-Qing Liang who is currently a postdoc with UMASS Medical School.

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Messenger RNA, which is a type of molecule, can be used to treat certain diseases caused by health problems in our genes. However, up until recently, it was hard to get this RNA into the right place in our body without any negative side effects. To solve this, researchers have been testing out using RNA-based treatments on liver conditions and some Covid-19 vaccines which are injected directly into muscle. In cases like these, the RNA is stuck inside a small sphere made of fats called a lipid nanoparticle so that its effects last for longer and goes to where it needs to go in the body.

A few years ago, Anderson’s laboratory tried to make particles which could penetrate the lung’s epithelial cells easily. In 2019 they finally made it by creating nanoparticles which carried mRNA (a biological material) with a special protein. These particles were made up of polymers instead of lipids so aerosolizing them into lungs was simpler. But more work is still needed to improve them and make them even better.

Researchers have designed small particles called lipid nanoparticles that can be directed towards our lungs. These particles are made up of molecules with two parts: a positively charged head and an extended fatty tail. This positive charge helps the particles attach to negatively charged mRNA – the stuff inside cells – allowing them to escape into cells.

The researchers studied how the special ‘lipid tail’ structure of particles helps it pass through cell membranes. They used 10 different chemical structures, paired with 72 headgroups in experiments involving mice. The results showed which combinations were most likely to reach the lungs.

Unlocking the Possibilities of Nanoparticle Delivery to Treat Lung Diseases

Researchers made a special particle that could be used to deliver the instructions for CRISPR/Cas9 into the lung cells of mice. The CRISPR/Cas9 was designed to “switch off” a stop signal that prevented a gene from turning on. When it did switch off, this gene created a fluorescent protein and made the cell “glow.” Researchers then measure how many cells “glowed” to see if the instructions were delivered properly.

The researchers discovered that after taking one dose, around 40 percent of lung cells were affected. When two and three dosage doses were taken, the level rose to over 50%, and 60% respectively. They also found that two types of epithelial cells called club cells and ciliated cells were affected at a rate of 15%.

Li says that the cells they edited are very important when it comes to respiratory diseases. Additionally, this special fatty substance can help get healthy messages into their lungs much better than any methods used until now.

The new particles that help deliver RNA quickly break down and disappear from the lungs in just a few days. That helps to prevent inflammation, and they can be given multiple times to patients if they need extra doses. This makes them better than using an altered form of an harmless virus called “adenoviruses”. Those viruses are really effective but can’t be used multiple times because our body won’t like it and will try to fight against it.

Dan Peer, the director of the Laboratory of Precision NanoMedicine at Tel Aviv University, says that this discovery could help with treatments for various lung diseases. This new platform has advantages over other conventional vaccines and therapies since it doesn’t need cells to work, can be created quickly, is really flexible and safe.

In this study, the researchers used a special method called “intratracheal instillation”, to give particles to people. This technique is often used for giving medicine through the lungs. Right now, they are trying to make their nanoparticles more stable so it can be blown out like air with a machine called nebulizer which helps to inhale them.

Researchers plan to use tiny particles to deliver mRNA (a special type of molecule) into the lungs. This could potentially correct a genetic mutation that is responsible for causing cystic fibrosis in mice. In addition, they also want to use it create treatments for other types of lung diseases like idiopathic pulmonary fibrosis and develop vaccines that would go directly to the lungs.

A group of scientists, lead by Bowen Li, Rajith Singh Manan, Shun-Qing Liang, Akiva Gordon, Allen Jiang, Andrew Varley and Guangping Gao with the help of Robert Langer and Wen Xue published a scientific article on Nature Biotechnology journal about how particles can be used to deliver messages to people’s lungs and change their genome. The article was titled “Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing” and it was published on March 30th 2023.

This research was paid for by many organizations, like Translate Bio, the National Institutes of Health, the Leslie Dan Faculty of Pharmacy startup fund, and the American Cancer Society. They all wanted to find out more about two things: 1) How gene therapy can help people with eye diseases; and 2) How a protein called “brain-derived neurotrophic factor” can treat retinitis pigmentosa.

Scientists are doing research to see how RNA interference and CRISPR/Cas9 technologies can be used in eye diseases. RNA interference may be able to help prevent something called posterior capsule opacification by blocking a certain pathway of signals in the body. Long noncoding RNA-MIAT is being studied to see how it affects lens epithelial to mesenchymal transition, which has been triggered by a substance called transforming growth factor-β2. Finally, CRISPR/Cas9 technology is being used to try and find out more about eye diseases.

1. A study about how both modern medicine and traditional Chinese medicine could help people with pulmonary fibrosis who had COVID-19 recover better.

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3. A computer process that uses an algorithm called hybrid differential evolution to deal with a difficult problem related to delivery, location, and inventory.

4. Using intelligence-based technology to classify and identify COVID-19 early on.

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