Old DNA May Lead to Hemophilia Therapy

University of Florida scientists say an ancient form of DNA found in fish might be useful in fighting hemophilia.

The researchers say the discovery brings them closer to finding a safe way to spur production of a missing protein in patients with the most common form of the hereditary bleeding disorder.

Using a dormant strand of DNA that’s existed in fish for millions of years, the researchers successfully replaced the faulty gene responsible for the disease in neonatal mice.

The degree to which these patients have problems from hemophilia stems from how much of this protein, factor VIII, is missing, said Dr. Bradley Fletcher, an assistant professor of pharmacology and one of the lead authors of the study. If they have very low levels of it, they have lifelong problems of bleeding…

More than 18,000 Americans, nearly all men, suffer from hemophilia A, the most common form of the disease. Currently, the only safe treatment for the disorder is a purified form of the protein, but it can cost patients thousands of dollars and its effects are short-lived.

The UF study is published online this month in the journal Molecular Therapy.