Existing drug preserves insulin production in new Type 1 diabetes cases, study finds
A drug used to help kidney transplant recipients ward off rejection of their new organs is showing promise in another area: preserving insulin production in newly diagnosed Type 1 diabetes patients. A TrialNet study led by UF Health researchers sought to determine whether the drug thymoglobulin, alone or in combination with another drug, could slow insulin loss. Clinical trial participants given a low dose of the medication, also known as ATG, had higher levels of insulin production compared with placebo recipients. The drug also reduced significantly reduced hemoglobin A1c levels, a long-term measure of average blood sugar levels. — Doug Bennett
New technique allows for earlier detection of faulty, repeating genetic sequences
UF Health researchers have found a way to detect faulty, repeating genetic sequences much sooner with a blood test. Key to the findings are introns, a part of genes generally not involved in protein formation. In one type of inherited form of ALS and other so-called repeat expansion disorders, mutant introns that are normally removed before protein production get left behind. For some of these disorders, the lingering mutant introns can be readily detected in tissue and white blood cells — creating a rapid and inexpensive way to detect repeat expansion disorders, the researchers found. — Doug Bennett
Gene therapy improves daylight vision, color vision deficiencies in animal model
For people with blue cone monochromacy, the world is blurry, colorless and uncomfortably bright. UF Health researchers have used a gene therapy to restore visual functions to affected cone photoreceptor cells during tests in mice. The disease is caused by defective genes that affect red and green cone photoreceptors in the retina, leaving patients with only blue color receptors. Researchers used an adeno-associated virus to deliver human genes into mutant mouse retinas. The treatment restored cone electroretinography in up to 70 percent of the mice with normal vision. — Doug Bennett
