Scientists at the University at Buffalo have made a groundbreaking discovery that could revolutionize our understanding of brain cell clumps associated with neurodegenerative diseases like Huntington’s and ALS.

For decades, researchers have struggled to understand how these solid-like clusters of RNA form in brain cells, making it challenging to develop effective treatments. The mystery was finally cracked when the University at Buffalo team uncovered that tiny droplets of protein and nucleic acids in cells contribute to the formation of RNA clusters.

But what’s even more remarkable is that the researchers not only figured out how these clusters form but also demonstrated a way to prevent and disassemble them using an engineered strand of RNA known as an antisense oligonucleotide (ASO).

“This is a major breakthrough,” said Priya Banerjee, PhD, associate professor in the Department of Physics at the UB College of Arts and Sciences. “We’re not only able to understand how these clusters form but also find a way to break them apart.”

The team’s study published in Nature Chemistry reveals that RNA-binding protein G3BP1 can prevent cluster formation by binding to sticky RNA molecules, while an ASO can disassemble the existing clusters. The researchers found that ASO’s disassembly abilities are highly tied to its specific sequence, suggesting it can be tailored to target specific repeat RNAs.

“This has significant implications for potential therapeutic applications,” Banerjee said. “We’re excited about the possibilities of using ASOs to develop new treatments for neurodegenerative diseases.”

Banerjee is also exploring RNA’s role in the origin of life, studying whether biomolecular condensates may have protected RNA’s functions as biomolecular catalysts in the harsh prebiotic world.

“It really just shows how RNAs may have evolved to take these different forms of matter, some of which are extremely useful for biological functions and perhaps even life itself – and others that can bring about disease,” Banerjee said.

The article “Flossing for Vaccines: A New Method to Deliver Immunizations” discusses a novel technique developed by researchers to deliver vaccines via dental floss. The method targets the junctional epithelium, a thin layer of tissue between the tooth and gum, which lacks barrier features and is more permeable than other epithelial tissues. This allows for enhanced antibody production across the body’s mucosal layers.

The researchers applied vaccine-coated floss to lab mice and compared antibody production in three different methods: via the junctional epithelium, nasal epithelium, or under the tongue. They found that applying vaccine via the junctional epithelium produced a superior antibody response on mucosal surfaces than the current gold standard for vaccinating via the oral cavity.

This technique has significant advantages beyond improved antibody response on mucosal surfaces. It is easy to administer and addresses concerns many people have about being vaccinated with needles. The researchers also believe this method should be comparable in price to other vaccine delivery techniques.

However, there are some drawbacks to consider. This technique would not work on infants and toddlers who do not yet have teeth. Additionally, the approach may not be suitable for people with gum disease or other oral infections, and more research is needed to fully understand its potential benefits and limitations.

The study was published in the journal Nature Biomedical Engineering and was supported by grants from the National Institutes of Health and funds from the Whitacre Endowed Chair in Science and Engineering at Texas Tech University. The researchers are optimistic about this work and may move toward clinical trials depending on their findings.

In a groundbreaking study, researchers at Mayo Clinic have discovered that a sugar molecule used by cancer cells to evade the immune system could also help treat type 1 diabetes. The team, led by immunology researcher Virginia Shapiro, Ph.D., found that dressing up beta cells with the same sugar molecule, known as sialic acid, enabled the immune system to tolerate them.

Type 1 diabetes is a chronic autoimmune condition in which the immune system mistakenly attacks pancreatic beta cells that produce insulin. This leads to an estimated 1.3 million people in the U.S. suffering from the disease. In their studies, Shapiro’s team used a cancer mechanism and turned it on its head by applying it to type 1 diabetes.

The researchers took a closer look at a preclinical model of type 1 diabetes and found that beta cells engineered to produce an enzyme called ST8Sia6, which increases sialic acid on the surface of tumor cells, were not attacked by the immune system. In fact, they were 90% effective in preventing the development of type 1 diabetes.

The team’s findings show that it is possible to engineer beta cells that do not prompt an immune response. This breakthrough has the potential to improve therapy for patients with type 1 diabetes, who currently rely on synthetic insulin or transplantation of pancreatic islet cells with immunosuppression.

Dr. Shapiro aims to explore using the engineered beta cells in transplantable islet cells without the need for immunosuppression. While still in the early stages, this study may be one step toward improving care for patients with type 1 diabetes.