For many years, scientists have envisioned a world where genetic disorders, like hemophilia—a condition affecting blood clotting—would no longer exist. The concept of gene therapy, which involves replacing faulty genes with healthy ones, has shown great potential. However, a significant challenge has been finding a reliable and safe method to introduce these genes. Recently, researchers achieved a major advancement in gene editing technology that could transform the treatment of genetic disorders.
For many years, scientists have envisioned a world where genetic disorders, like hemophilia—a condition affecting blood clotting—would no longer exist. The concept of gene therapy, which involves replacing faulty genes with healthy ones, has shown great potential. However, a significant challenge has been finding a reliable and safe method to introduce these genes.
Researchers from the University of Hawai’i’s John A. Burns School of Medicine (JABSOM) have developed an innovative gene editing technique that might change the way we approach genetic disorders. Their novel method offers a speedier, safer, and more effective way to integrate healthy genes into the body, which could lead to potential treatments for numerous medical conditions. Their findings were published recently in Nucleic Acids Research.
Here’s a brief overview of the process.
While existing methods can correct gene errors, they often inadvertently cause harm by breaking the DNA. Moreover, these techniques tend to have difficulty inserting larger segments of genetic material, such as entire genes.
The new approach, created by Dr. Jesse Owens and his team—comprising Dr. Brian Hew, Dr. Ryuei Sato, and Sabranth Gupta from JABSOM’s Institute for Biogenesis Research and Cell and Molecular Biology Department—overcomes these challenges. They utilized laboratory evolution to produce a highly efficient integrase that can insert therapeutic genes into the genome with remarkable success.
“It’s akin to having a ‘paste’ function for the human genome,” explained Dr. Owens. “It employs specially designed ‘integrases’ to insert healthy genes precisely where needed, without causing DNA breaks. This technique shows much higher efficiency, achieving success rates of up to 96% in certain scenarios.”
“This could enable quicker and more cost-effective treatments for a variety of diseases, potentially affecting hundreds of conditions linked to a single faulty gene,” Dr. Owens added.
Accelerating Treatment Development and Expanding Potential Applications
The significance of this research goes beyond gene therapy. The capability to insert large DNA segments efficiently can benefit various medical fields.
In the process of creating cell lines for therapeutic protein production, the gene that encodes the protein is typically inserted haphazardly into the genome, resulting in suboptimal production locations. This is akin to looking for a needle in a haystack. Moreover, identifying a cell that correctly integrates the gene and produces the desired protein can take a considerable amount of time.
Instead of the tedious process of searching, Dr. Owens’ technique creates a ‘stack of needles’ by directing the gene exactly where it needs to be, which can drastically expedite the development timeline.
“JABSOM is proud to foster innovative researchers like Jesse Owens, whose work has the potential to make a worldwide difference,” commented Sam Shomaker, dean of the University of Hawai’i John A. Burns School of Medicine. “This research, conducted in our lab in the heart of the Pacific, could significantly enhance how we care for genetic diseases.”
Dr. Owens and his team are investigating how this novel method could hasten the development and production of biologics and advanced therapies, such as antibodies. Currently, finding the appropriate cell line for efficient production can take a long time. However, the new genome engineering tool Dr. Owens developed can shorten the timeline for developing cell lines and hasten the production of essential therapeutics.
Additionally, Dr. Owens has established a nonprofit organization aimed at promoting local genetic engineering research in Hawaii.
