LAB Notes

Gene therapy shows promise against Pompe

University of Florida researchers have successfully used gene therapy to treat patients with infantile onset Pompe disease, a progressive condition that compromises cardiopulmonary function in the first years of life. The team at UF’s Powell Center for Rare Disease Research and Therapy conducted the first in-human study of gene therapy to treat respiratory dysfunction in patients with Pompe. In the study, which began in 2011, an adeno-associated virus vector was used to carry a functional copy of the affected gene to muscle cells in the diaphragms of nine patients. In results recently published online in Experimental Neurology, breathing function improved with gene therapy, compared to treatment with breathing exercises. — Mindy Cameron

Another dangerous virus found lurking in Haiti

University of Florida researchers have identified a boy in Haiti with Mayaro virus, a serious mosquito-borne illness that has never been reported in the Caribbean nation. Faculty from the UF Emerging Pathogens Institute were in the region during and after the 2014 chikungunya outbreak and obtained plasma samples from febrile children, which they analyzed for the chikungunya virus RNA using a genetic identification technique known as reverse transcription polymerase chain reaction. UF’s Maha Elbadry, Ph.D., sent the samples from Haiti to the EPI for additional virology and molecular analyses. One boy was found to have the dengue virus and a different virus that was subsequently identified as Mayaro. The findings were published online in the Centers for Disease Control and Prevention’s journal Emerging Infectious Diseases. — Evan Barton

Deep dive in developing brains

How does sleep affect brain development in adolescents? What about screen time, sports-related head injuries, marijuana use? The University of Florida and 18 other institutions will examine those and other factors under the Adolescent Brain Cognitive Development, or ABCD, study — the largest long-term study of brain development and child health ever undertaken in the United States. Funded by the National Institutes of Health, the study will follow the biological and behavioral development of 10,000 children – about 400 in the Gainesville region – from ages 9 and 10 into early adulthood. A $3.76 million grant to UF allows its Evelyn F. and William L. McKnight Brain Institute to participate, said Sara Jo Nixon, Ph.D., co-principal investigator at the UF Health site with Linda B. Cottler, Ph.D., M.P.H., founding chair of UF’s department of epidemiology. —Michelle Koidin Jaffee