News Column

Recent Studies from Washington University Add New Data to Gene Therapy

January 30, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on Biotechnology. According to news reporting out of St. Louis, Missouri, by NewsRx editors, research stated, "Canine models have many advantages for evaluating therapy of human central nervous system (CNS) diseases. In contrast to nonhuman primate models, naturally occurring canine CNS diseases are common."

Our news journalists obtained a quote from the research from Washington University, "In contrast to murine models, the dog's lifespan is long, its brain is large and the diseases affecting it commonly have the same molecular, pathological and clinical phenotype as the human diseases. We compared the ability of four intracerebrally injected adeno-associated virus vector (AAV) serotypes to transduce the dog brain with green fluorescent protein as the first step in using these vectors to evaluate both delivery and efficacy in naturally occurring canine homologs of human diseases. Quantitative measures of transduction, maximum diameter and area, identified both AAV2/9 and AAV2/rh10 as significantly more efficient than either AAV2/1 or AAV2/5 at transducing cerebral cortex, caudate nucleus, thalamus and internal capsule. Fluorescence co-labeling with cell-type-specific antibodies demonstrated that AAV2/9 and AAV2/rh10 were capable of primarily transducing neurons, although glial transduction was also identified and found to be more efficient with the AAV2/9 vector."

According to the news editors, the research concluded: "These data are a prerequisite to evaluating the efficacy of recombinant AAV vectors carrying disease-modifying transgenes to treat naturally occurring canine models in preclinical studies of human CNS disease therapy."

For more information on this research see: Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain. Gene Therapy, 2014;21(1):28-36. Gene Therapy can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group -; Gene Therapy -

Our news journalists report that additional information may be obtained by contacting G.P. Swain, Washington University, Sch Med, Dept. of Genet, St Louis, MO 63110, United States. Additional authors for this research include M. Prociuk, J.H. Bagel, P. O'Donnell, K. Berger, K. Drobatz, B.L. Gurda, M.E. Haskins, M.S. Sands and C.H. Vite (see also Biotechnology).

Keywords for this news article include: Biotechnology, Cells, Neurons, Therapy, Viruses, Missouri, Virology, St. Louis, United States, North and Central America

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Source: Gene Therapy Weekly

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