Treatment advances are occurring daily in the field of rare disease. As researchers hone their strategies and tools, new technologies are emerging with the potential to significantly impact patients with conditions like cystic fibrosis or idiopathic pulmonary fibrosis (IPF). In fact, shares Science Daily, the development of nanoparticles that can deliver gene editing directly to the lungs could offer an easier, inhalable treatment for lung diseases. Engineers from the University of Massachusetts Medical School and the Massachusetts Institute of Technology (MIT) recently designed nanoparticles to achieve this goal.
What are Nanoparticles?
Before we dive into these messenger RNA (mRNA)-delivering nanoparticles, let’s first get a basic overview of what nanoparticles actually are. TWI Global explains that nanoparticles are small particles, undetectable by the human eye, that are between 1-100 nanometers in size. Further, shares TWI Global:
Nonparticles can exhibit significantly different physical and chemical properties to their larger material counterparts.
Nanoparticles have been used in multiple industries and situations, such as:
- Treating polluted water
- Inclusion in sunscreen to offer longer UV protection
- Creating aircraft wings or other aerospace material
- The development of stronger camouflaging in military uniforms
- Drug delivery directly to the disease source or tumor
Delivering mRNA Treatments
Given the wide applicability of nanoparticles within healthcare, it’s no surprise that the research team looked to nanoparticles as potential therapeutic interventions for lung diseases. In their study, published in Nature Biotechnology, the researchers hoped to overcome issues with drug delivery when administering genomic medicine to the lungs. It can often be difficult to administer mRNA therapies without what Science Daily refers to as “off-target effects.” Finding ways to deliver mRNA therapies directly and safely could be significantly beneficial.
For this study, the research team developed lipid nanoparticles that specifically targeted the lungs. These nanoparticles have a lipid tail structure that helps them to pass through cell membranes, as well as a positively charged headgroup so that mRNA could escape from cell structures. Combinations including 72 headgroups and 10 lipid tails were evaluated in mice models. Through this, the research team determined which combination(s) were most likely to be effective in reaching and treating the lungs.
By adding gene editing technology (CRISPR/Cas9) to the nanoparticles, the therapy could be delivered directly to lung cells. Researchers removed a “stop” signal in the lungs and turned on a fluorescent protein. Evaluating how far this protein has spread provides more insight into how effective the nanoparticles were in prompting mRNA expression. They found that mRNA was expressed from 15-60% depending on the specific cell types. mRNA expression increased with multiple nanoparticle doses.
While more research is needed to test this potential therapeutic solution further, and to create an aerosolized version for easier administration, this does show promise for the future treatment of rare or uncommon lung diseases.