Scientists have developed a new nanoparticle that can carry gene-editing technology into the lungs of mice in an effort to develop life-changing treatments for genetic lung diseases such as cystic fibrosis.
Messenger RNA (mRNA), the therapeutic gene agent being used to treat said genetic lung diseases, needs to travel directly to the cells in order to work – and scientists at the Massachusetts Institute of Technology (MIT) hope their new nanoparticles will help boost successful treatment.
Dr. Joseph Ambani, doctor at Narok County Hospital, said: “Nanoparticles enhance the delivery of drugs. In the case of lung disease, it can ensure therapeutic agents, such as gene therapies and drugs reach the affected lung cells.”
“This can make gene therapies or drugs more effective and keep any side effects to a minimum.”
A group of scientists at MIT University have created what they call lipid nanoparticles – which they say are able to target the lungs and deliver the important gene-altering mRNA.
The scientists developed the new lipid nanoparticle with nanoparticles made up of a positively charged headgroup, which allows the particles to interact with the negatively charged mRNa, along with a lipid tail that helps the particles to move through the membrane and into the cell, NewAtlas reported.
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After painstakingly experimenting with over 70 headgroups and 10 tails, the scientists managed to crack the nanoparticle that was able to reach the lungs.
“This lipid can enable us to deliver mRNA to the lung much more efficiently than any other delivery system that has been reported so far,” said Bowen Li, lead author of the study.
The researchers found that using lipid nanoparticles was a much more effective vehicle than the adeno-associated virus (AAV), which is also used to deliver gene therapy.
According to the researchers, AAV causes the body to produce an immune response, meaning it can’t be used on the same person several times. However, lipid nanoparticles do not produce an immune response – so can be used as many times as they’re needed.
The MIT University team is hoping to make the lipid nanoparticles even more stable so they can be administered via a nebuliser, which administers drugs into the lungs via a mist, and not just a vaccine.
Daniel Anderson, corresponding co-author of the study said: “This is the first demonstration of a highly efficient delivery of RNA to the lungs in mice.
“We are hopeful that it can be used to treat or repair a range of genetic diseases, including cystic fibrosis.”