2024-08-29 18:15:00
Gene therapy is changing the way we approach diseases
The idea of replacing defective genes with healthy genes has been very promising. But a major hurdle has been finding a safe and efficient way to get those genes.
Researchers at the University of Hawaii John A. Burns School of Medicine (JABSOM) have made a major breakthrough in multiple editing technology. Their new method offers a faster, safer and more effective way to introduce healthy genes into the body, which could lead to treatments for hundreds of diseases.
Current methods can correct errors in genes, but they can also cause unwanted damage by creating breaks in DNA. In addition, they are difficult to insert many genetic materials, such as whole genes. The new technique, developed by the Biogenesis Research Center and the Department of Cellular and Molecular Biology at JABSOM, addresses these limitations. They used laboratory evolution to generate a new superactive fusion capable of inserting therapeutic genes into the genome with transcription.
The implications of this research go beyond gene therapy. The ability to efficiently insert large fragments of DNA has applications in other areas of medicine. When cell lines are created to produce therapeutic proteins, the gene that encodes the protein is often inserted randomly into the genome and is rarely placed in a position in the genome that is suitable for production. Finding a cell with the correctly inserted gene and producing the desired protein can take several months.
The new technique creates a bunch of needles and sends the gene directly to the desired location, speeding up the growth process significantly.
The team is exploring how this process can accelerate the development and production of biologics and advanced treatments such as antibiotics. Currently, finding the right cell line for efficient production can be a time-consuming process. However, new genomic engineering tools can reduce cell line development time and accelerate the development of life-saving treatments.
This study was published in the journal Nucleic Acids Research.
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