New technology could safely treat hundreds of genetic diseases

Gene therapy has long been a source of hope for the millions of people affected by genetic diseases, offering the promise of replacing faulty genes with healthy ones to treat conditions like the life-threatening blood clotting disorder hemophilia.

Despite its potential, the challenge has always been finding a safe, effective, and efficient method for delivering these therapeutic genes into the body. Now, a major breakthrough in gene-editing technology is poised to transform this dream into reality.

Researchers at the University of Hawai’i’s John A. Burns School of Medicine (JABSOM) have developed a new technique that ensures quicker, safer, and highly efficient delivery of healthy genes, potentially offering treatment for hundreds of genetic conditions.

Out with the old, in with the new

Existing gene-editing techniques, while capable of rectifying errors in genes, potentially endanger the DNA by causing unintended breaks. Moreover, they falter when it comes to the insertion of larger genetic segments, including entire genes.

The new method developed by Dr. Jesse Owens and his team overcomes these limitations by using a novel super-active integrase to insert therapeutic genes without breaking DNA, achieving success rates as high as 96 percent.

“It’s like having a ‘paste’ function for the human genome,” said Dr. Owens. “It uses specially engineered ‘integrases’ to carefully insert healthy genes into the exact location needed, without causing breaks in the DNA. This method is much more efficient, with success rates of up to 96% in some cases.”

According to Dr. Owens, this technology could lead to faster and more affordable treatments for a wide range of genetic diseases, potentially impacting hundreds of conditions with a single faulty gene.

Beyond gene therapy

The implications of the research extend beyond the scope of gene therapy. The ability to efficiently insert large DNA fragments significantly speeds up the development process, opening up exciting possibilities in various other medical sectors.

When creating cell lines for therapeutic protein production, the gene encoding the protein is usually randomly inserted into the genome, making the process like “searching for a needle in a haystack.”

This often results in a time-consuming and inefficient process. Instead, Dr. Owens’ technique “makes a stack of needles,” delivering the gene directly to the desired location, thereby accelerating the development process.

This advancement could revolutionize how we produce biologics and advanced therapies like antibodies.

Creating a global impact, one gene at a time

“JABSOM takes pride in nurturing talented researchers like Jesse Owens, whose work has the power to create a global impact,” said Dr. Sam Shomaker, dean of the University of Hawai’i John A. Burns School of Medicine.

The genetic engineering research led by Dr. Owens’ team in their Pacific-based lab is set to significantly enhance our approach to treating genetic disorders.

The researchers are also exploring how this avant-garde technique can expedite the development and manufacturing of biologics and advanced therapies.

Existing methods for identifying efficient cell lines for production can be time-consuming. However, the newly developed genome engineering tool could reduce the cell line development timeline, accelerating the production of life-saving therapeutics.

To further promote the advancement of genetic engineering, Dr. Owens has established a 501c3 nonprofit organization dedicated to supporting local research in Hawaii. This initiative emphasizes the importance of his work and its potential to revolutionize the future of genetic medicine.

The future of gene therapy

As the experts continue to refine this innovative approach, the future of gene therapy looks promising.

The new method developed by Dr. Owens and his team not only enhances the safety and efficiency of gene editing but also opens up a world of possibilities for other medical applications.

By precisely inserting therapeutic genes without causing unintended DNA damage, this breakthrough can pave the way for treatments that were previously deemed too risky or challenging.

The potential of this technology goes beyond curing genetic diseases. It could revolutionize the production of therapeutic proteins, advance the development of advanced cell therapies, and even accelerate vaccine development.

With such wide-ranging applications, the advancements in gene therapy technology could significantly impact healthcare, making personalized medicine more accessible and effective for patients worldwide.

The study is published in the journal Nucleic Acids Research.

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