Australian scientists have developed a groundbreaking experimental drug that could be used to treat a range of diseases, from diabetes and heart disease to cancer.
Researchers at the University of New South Wales (UNSW) have introduced a universal therapy that has been shown to be highly effective in treating various skin cancers. Named Dz13, this drug can be utilized to address vision loss caused by age-related macular degeneration, treat heart disease victims, and combat infections.
Published in the journal Nature Biotechnology, this therapy targets a disease-causing gene known as c-Jun, a key regulatory machine found in diseased blood vessels, eyes, joints, and intestines. Molecular biologist Levon Khachigian, who led the study, described the drug as a “molecular assassin”—a DNA molecule with a blade attached—designed to seek out the disease-causing gene and cut it in two.
Representing a new generation of gene therapies, this treatment is the first calculated to eliminate the offending gene. As a result, a series of other genes typically controlled by the main regulatory machine will never be activated, preventing the onset of disease.
The new drug has shown effects on cancer tumors and skin cancer in preclinical trials primarily by cutting off their blood supply. Animal trials have yielded “promising results.” Next year, this drug will be tested on 10 adults without melanoma skin cancer.
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