Gene therapy has shown great potential for treating various genetic disorders. However, its efficacy is limited by the inability to deliver therapeutic genes to specific cells without causing toxicity or immune response. One promising solution is the use of tiny bubbles, called “microbubbles,” which can transfer genes to targeted cells safely and effectively. In this article, we explore the science behind microbubble-mediated gene therapy and its potential applications in treating genetic disorders.
Researchers at Case Western Reserve University School of Medicine in Cleveland, Ohio, are developing a process that uses nanobubbles to deliver Messenger ribonucleic acid (mRNA) to specific cells in the body. mRNA is a genetic material that tells the body how to make protein, and is used in vaccines such as Moderna’s Covid-19 vaccine. The researchers are working to develop a more efficient way to deliver the material into targeted cells using nanobubbles, which are gas-core particles that are 1,000 times smaller than microbubbles and can escape from leaky blood vessels in diseases such as tumours, and accumulate in the targeted tissue. This is expected to be especially important in treating diseases such as cancer or genetic disorders, where specific cells need to be targeted without harming healthy cells.
The nanobubbles are combined with noninvasive, gentle sound waves that can be used without harming the body, and the nanobubbles make it easier to enter cells by “cavitation,” or popping the bubbles, resulting in more efficient gene delivery. Moderna awarded postdoctoral scholar Pinunta “Petch” Nittayacharn in the Exner lab the fellowship for their work in the drugmaker’s inaugural round of research grants.
“If these challenges can be overcome, this approach could lead to new and more effective ways of delivering therapies to specific cells in the body,” said Agata Exner, the Henry Willson Payne Professor and vice chair for basic research in the Department of Radiology at the School of Medicine, who is leading the research. However, the researchers did acknowledge that there are some challenges and potential limitations to this approach that will need to be addressed, such as optimizing the design of the nanobubbles, ensuring that the mRNA is effectively released from the nanobubbles once inside the cell, and minimizing any potential toxicity or immune reactions.
The future of mRNA delivery will be shaped by this and many other approaches being developed in research labs worldwide, said Agata Exner.
Overall, this technology shows promise in creating new and effective ways to deliver therapies to specific cells in the body, and has the potential to transform the treatment of diseases such as cancer and genetic disorders.
In conclusion, scientists are making strides in improving gene therapy by using tiny bubbles. These bubbles can deliver genetic material to target cells, while also enhancing the effectiveness of the therapy. In addition to gene therapy, the use of nanobubbles has also shown promise in various medical applications such as drug delivery, tissue regeneration, and cancer treatment. As research in this field continues to grow, it is clear that tiny bubbles have the potential to revolutionize the way we approach healthcare and medicine.
