Breakthrough in Gene Therapy Promises Safer Treatment for Genetic Diseases

Researchers at the University of Hawai’i’s John A. Burns School of Medicine have unveiled a groundbreaking gene-editing method that promises safer and more effective treatment for genetic diseases, earth.com reported.

This innovative technique utilizes a super-active integrase to insert therapeutic genes into the genome with a remarkable success rate of up to 96%, eliminating the risk of DNA damage associated with traditional methods.

The new approach not only enhances gene therapy but also significantly accelerates the production of biologics and advanced therapies, including antibodies. By allowing for the precise insertion of large DNA fragments, the technique streamlines the development of efficient cell lines for therapeutic protein production, transforming a previously time-consuming process.

The implications of this research extend to various medical applications, including the development of advanced cell therapies and vaccines. This advancement marks a significant step forward in making personalized medicine more accessible and effective for patients suffering from genetic disorders. The findings are detailed in the journal Nucleic Acids Research.