A team of researchers at the University of Connecticut is collaborating to develop new asthma therapeutics using gene-silencing nanocapsules in order to help patients who are not benefiting from existing treatments, including steroid-based inhalers.
Lead researcher Prof. Jessica Rouge said, “When treating asthma, many people think of small molecule anti-inflammatory medications as the way to go, but there are plenty of patients who have asthma who do not respond to corticosteroids. There’s an unmet need for creating different therapeutics that can suppress asthma for this group of people.”
The researchers published their findings in the journal ACS (American Chemical Society) Nano.
The group designed nanomaterials and targeted therapeutics that deliver gene silencing messages to cells, according to Science Daily.
The authors explained that a nucleic acid nanocapsule (NAN) is designed to selectively deliver an enzyme, known as DNAzyme, to silence a component of the immune response that leads to the over-expression of immune components that play a significant role in allergic asthma attacks.
Co-lead researcher Prof. Steven Szczepanek explained that this technology is specifically designed to treat allergic asthma, which constitutes about 50% of cases in adults and 90% in children.
Prof. Rogue said, “When using nanomaterials, we try to administer the therapy in a way that could allow us to use less materials to get a bigger effect.”
“First, we synthesize something called a surfactant, it’s much like soap and essentially forms a nanoscale bubble,” she explained. “Then we modify the surface chemistry of this bubble so it can conjugate or connect to DNA. The next step, and what’s unique to our lab, is we use enzymes to build the next piece to attach the DNA sequence that essentially cleaves mRNA encoding GATA-3.”
“We showed these gene-silencing sequences were effectively delivered using our formulation and we saw that they knocked down the gene target of interest,” Prof. Rogue added. “That was an exciting first step.”
Prof. Szczepanek said, “I thought this gene silencing technology was a fantastic application for an asthma therapeutic.”
Prof. Rouge said they are hopeful to get NIH funding to continue the research to move onto the next step. She said, “We want to figure out, Where do these nanocapsules go? We need to do a biodistribution study and other logical next steps, like pharmacokinetics and determining how long these therapeutics last in an organism.”
The researchers have recently been awarded a patent for the nanocapsule formulation, and soon they hope to commercialize it. Prof. Szczepanek hinted that novel technology could be delivered via an inhaler, just like the current asthma medications.
