better than the standard liquid formulation when it came to transferring gene therapy components to cells in laboratory studies. This discovery has potential implications for the delivery of gene therapies, as foaming medications are already being used to treat various conditions such as varicose veins, hemorrhoids, skin wounds, and hair loss. The study, which was published in Nature Communications on May 28, highlighted the potential of using foam as a vehicle for delivering expensive gene therapies. Bioengineer Matthias Stephan, MD, PhD, led the Fred Hutch team in conducting this research.The gene therapy foam developed by Professor Stephan from Fred Hutch’s Translational and Therapeutics Division has proven to be more effective than the traditional liquid formulation in transferring gene therapy components to cells in laboratory studies. This innovation is particularly significant as gene therapies are known to be costly and challenging to produce. By utilizing a small amount of the expensive gene therapy drug and increasing its volume through embedding it in a solution predominantly consisting of densely packed air bubbles, the foam has demonstrated the ability to safely and effectively transfer gene therapy agents to cells. This development marks a significant advancement in the field of gene therapy.New therapies for various diseases like cancers, infectious diseases, and inherited disorders show great potential for offering effective and long-term treatments by addressing the root causes of the conditions. Nevertheless, these therapies encounter obstacles such as the challenge of delivering them to the specific parts of the body that require treatment, as well as the high expenses associated with producing modified viruses called vectors, which are used to transport the therapies to diseased cells. Foam may hold the answer to simplifying and reducing the cost of delivering gene therapies.
While the research is preliminary, Stephan believes that the gene therapy foam could be a viable option for treating cancers located in restricted areas.
There are certain diseases that affect the digestive system, such as ovarian, pancreatic, and gastrointestinal cancer, as well as autoimmune diseases.
Researcher Stephan mentioned that the foam could potentially be used to target body tissues affected by different diseases and deliver medication directly to those diseased cells. The foam would then be able to drain away, leaving the gene therapy behind.
Prior to this development, Stephan had used other materials such as nanoparticles and tiny implantable sponges to deliver genetic instructions to combat diseases at the cellular level. His inspiration for the foam delivery method came from shaving foam.
Experiments in the lab
Stephan and his team conducted a series of laboratory experiments to see if they could combine a small amount of expensive gene therapy vectors with gene editing materials and various foaming agents. They then applied this mixture to cells to observe whether the cells would take up the gene edits.
To test their approach, tThe team utilized a different type of immune-based treatment: the mRNA for a COVID-19 vaccine. This vaccine instructs the immune system to fight the virus by presenting the biological code of the virus to the immune system.
The Fred Hutch team created their own version of a COVID-19 mRNA vaccine with a biological modification that made it possible to glow once it entered cells. This allowed the team to observe the vaccine under a microscope once it was taken up by cells.
In order to create the foam, the team discovered that methylcellulose, a food additive commonly found in ice cream and other products, was the best foam for transferring gene therapy.The therapy product was designed to effectively deliver gene therapy to cells and remain in place once deposited. When combined with cells, the foam significantly improved gene transfer and performed better than liquid formulations. In a study with mice, injection of a small amount of foam into the abdominal cavity resulted in the transfer of gene therapy to targeted cells within one day, without causing significant side effects. Although still far from being used in humans, the researcher aims to collaborate with external academic and industry partners to further develop the technology.The aim of my work is to enhance the effectiveness of modern medications for people all over the world,” Stephan stated. He believes that our future advancements in medicine will come from a deep understanding of disease biology and our ability to develop innovative solutions in unexpected places.”
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