Professor Ke Zhang working in his lab.

Researcher uses gene regulation to improve drug delivery and treatment of rare genetic diseases

Duchenne Muscular Dystrophy is one of the most severe forms of muscular dystrophy. It’s also one of the most rare, as the genetic disease affects one in 3,500 to 5,000 newborn boys. For Ke Zhang, a professor of chemistry and chemical biology at Northeastern University, that’s still one too many.

As part of his Spark Fund awarded research, Zhang is working on a novel form of drug delivery approach for improved gene regulation that could change the status quo. Helmed by pacDNA LLC, Zhang’s Center for Research Innovation-supported spinout, Zhang’s approach to gene regulation is built on oligonucleotides. These small but mighty strands of DNA-like molecules, along with a carrier called bottlebrush polymer, could be the trick to targeting deadly genetic diseases like DMD and cancer.

Traditionally, drugs consist of small molecules that impact specific proteins in the body, preventing a protein from working or activating a protein to make it work better. Gene regulation techniques go directly to the source, affecting the mRNA encoding the protein so that the problematic proteins are never created in the first place. The method Zhang is using, called exon skipping, goes one step further, causing cells to skip over faulty sections of the genetic code, called exons, leading to the production of functional proteins.

“For the exon skipping approach, we try to correct the mistake in the mRNA so the correct proteins can be produced,” Zhang says. “That’s where our oligonucleotide therapy operates.”

Read more from Northeastern Global News.

Photo by Alyssa Stone/Northeastern University.

Chemistry and Chemical Biology