Advancements in Gene Therapy and mRNA Delivery for Lung Diseases
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| Improving gene therapy for lung disease |
Scientists at The Ohio State University College of Pharmacy have developed an inhalable nanoparticle that can be used to deliver messenger RNA, the technology powering COVID-19 vaccinations, to cells in cell culture or to the lungs of patients in mouse models. We have developed a unique approach to aerosolize.
According to the researchers, current nanoparticle nebulization techniques impose shear stress on the nanoparticles, impairing their ability to encapsulate genetic material and causing them to clump in certain areas of the lungs instead of being evenly distributed. The discovery is said to be important.
Sahay's group is investigating lipid nanoparticles (LNPs) as gene delivery vehicles. They focus specifically on cystic fibrosis, a degenerative genetic disease that affects 30,000 people in the United States and causes chronic lung infections. Approximately 1,000 new infections are discovered each year. The disease is caused by a defective gene called CFTR (cystic fibrosis transmembrane conductance regulator) and is characterized by dehydration of the lungs and the buildup of mucus that narrows the airways.
Lipids are compounds with fatty tails. These are found in many natural oils and waxes. Nanoparticles are tiny chunks of matter that range in size from one billionth of a meter to a thousandth of a meter. Messenger RNA sends instructions to cells to produce specific proteins.
In coronavirus vaccines, mRNA released by lipid nanoparticles instructs cells to produce a harmless fragment of the viral spike protein, triggering an immune response from the body. When treating cystic fibrosis, genetic material corrects mutations in the patient's CFTR gene.
Four years ago, Sahay said, he received a call from Oregon company Rare Air Health about the possibility of using microfluidic technology to aerosolize and deliver lipid nanoparticles.
Microfluidics is the study of the behavior of fluids as they flow through or are contained within microscopic devices with channels or chambers. Microscale fluids are dominated by surface forces rather than volume forces, and behave very differently than in everyday life.
Sahay added, "When Rare Air came to me, I thought this device would work best for our purposes. Extensive research has since been done and it is now in clinical use. We have demonstrated the superiority of this device in generating aerosolized nanoparticles compared to vibrating mesh nebulizers,” he added. The device does not allow nanoparticle aggregation and can deliver mRNA with greater precision than existing technologies. Another great thing is that the device can be controlled digitally, and Rare Air is developing a prototype for human use. ”
Oregon State researchers Julia Agelis, Jung-hwan Kim, Anthony Jozier, and Elissa Bloom contributed to the study along with Sahay. Scientists from Funai Microfluidic Systems in Lexington, Kentucky, also participated in the collaboration.
"Funai is focused on inkjet technology and large-scale manufacturing of these chips. They collaborated closely to create equipment suitable for aerosolization." "We demonstrate the relevance of novel devices and formulation sciences that have the potential to provide significant benefits," Sahay concluded. The study was funded by the National Heart, Lung, and Blood Institute and Rare Air.
According to the researchers, current nanoparticle nebulization techniques impose shear stress on the nanoparticles, impairing their ability to encapsulate genetic material and causing them to clump in certain areas of the lungs instead of being evenly distributed. The discovery is said to be important.
Sahay's group is investigating lipid nanoparticles (LNPs) as gene delivery vehicles. They focus specifically on cystic fibrosis, a degenerative genetic disease that affects 30,000 people in the United States and causes chronic lung infections. Approximately 1,000 new infections are discovered each year. The disease is caused by a defective gene called CFTR (cystic fibrosis transmembrane conductance regulator) and is characterized by dehydration of the lungs and the buildup of mucus that narrows the airways.
Lipids are compounds with fatty tails. These are found in many natural oils and waxes. Nanoparticles are tiny chunks of matter that range in size from one billionth of a meter to a thousandth of a meter. Messenger RNA sends instructions to cells to produce specific proteins.
In coronavirus vaccines, mRNA released by lipid nanoparticles instructs cells to produce a harmless fragment of the viral spike protein, triggering an immune response from the body. When treating cystic fibrosis, genetic material corrects mutations in the patient's CFTR gene.
Four years ago, Sahay said, he received a call from Oregon company Rare Air Health about the possibility of using microfluidic technology to aerosolize and deliver lipid nanoparticles.
Microfluidics is the study of the behavior of fluids as they flow through or are contained within microscopic devices with channels or chambers. Microscale fluids are dominated by surface forces rather than volume forces, and behave very differently than in everyday life.
Sahay added, "When Rare Air came to me, I thought this device would work best for our purposes. Extensive research has since been done and it is now in clinical use. We have demonstrated the superiority of this device in generating aerosolized nanoparticles compared to vibrating mesh nebulizers,” he added. The device does not allow nanoparticle aggregation and can deliver mRNA with greater precision than existing technologies. Another great thing is that the device can be controlled digitally, and Rare Air is developing a prototype for human use. ”
Oregon State researchers Julia Agelis, Jung-hwan Kim, Anthony Jozier, and Elissa Bloom contributed to the study along with Sahay. Scientists from Funai Microfluidic Systems in Lexington, Kentucky, also participated in the collaboration.
"Funai is focused on inkjet technology and large-scale manufacturing of these chips. They collaborated closely to create equipment suitable for aerosolization." "We demonstrate the relevance of novel devices and formulation sciences that have the potential to provide significant benefits," Sahay concluded. The study was funded by the National Heart, Lung, and Blood Institute and Rare Air.
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