UF teams with company to advance gene therapy treatments
A biotechnology company has raised $30 million to advance gene therapy treatments developed at the University of Florida Powell Gene Therapy Center. The funding also will help UF Health researchers bring a novel form of gene therapy to patients with Pompe disease, a rare form of muscular dystrophy.
Gene therapy study produces promising results for patients with hemophilia B
A fraction of patients with a common form of the bleeding disorder hemophilia develop an allergic reaction to the blood-clotting treatment they need to keep them alive. But using gene therapy, University of Florida researchers were able to reverse this reaction and provide long-lasting treatment for the disease in an animal model, according to findings published today in the journal EMBO Molecular Medicine.
If successful in humans, gene therapy could not only provide much-needed therapy for patients with hemophilia B, but also spare them from costly and difficult treatment regimens, said Roland Herzog, Ph.D., a professor of pediatrics in the UF College of Medicine and a senior author on the paper.
Satyanarayan Hegde, M.B.B.S., M.D., M.R.C.P.C.H. has new technology available for licensing
Dr. Hegde’s technology: Airway Oscillation Device for Preventing Mucus Obstructions in Cystic Fibrosis Patients’ Respiratory Tracts, helps remove mucus to prevent dangerous obstructions and make easier to obtain sputum samples for diagnosing lung infections.
Healthy lungs continually produce a small amount of mucus to retain moisture and trap foreign substances, such as bacteria and dust particles. Several conditions, including respiratory tract infections and cystic fibrosis, create too much mucus of abnormal consistency. An inherited disease that shortens life expectancy to less than 40 years, cystic fibrosis clogs vital organs with thick mucus. It affects approximately 30,000 people in the United States alone. Available treatments include physiotherapy and chest percussion therapy, which mechanically break down the mucus. These treatments, however, are not appropriate for patients who have brittle or broken bones, intense pain, asthma, tuberculosis, or injuries to the neck, spine, lungs or chest. University of Florida researchers have created a portable, battery-operated device that clears mucus buildup without the need for a vibrating/percussive vest. The device is safe for children as well as
the elderly and clinicians can easily modify its settings to meet specific treatment criteria, which is not possible with conventional external percussion devices.
- Appropriate for patients of any age or health condition, widening the potential market
- Can be self-administered as a facemask or mouthpiece, making it less expensive than percussive vests
- Runs on batteries, enhancing portability
- Allows clinicians to modify frequency and amplitude, increasing treatment flexibility compared to external percussion devices