News Column

Research Conducted at University of Naples Federico II Has Provided New Information about Gene Therapy

February 6, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Researchers detail new data in Biotechnology. According to news reporting from Naples, Italy, by NewsRx journalists, research stated, "Gene transfer to both cone and rod photoreceptors (PRs) is essential for gene therapy of inherited retinal degenerations that are caused by mutations in genes expressed in both PR types. Vectors based on the adeno-associated virus (AAV) efficiently transduce PRs of different species."

The news correspondents obtained a quote from the research from the University of Naples Federico II, "However, these are predominantly rods and little is known about the ability of the AAV to transduce cones in combination with rods. Here we show that AAV2/8 transduces pig cones to levels that are similar to AAV2/9, and the outer nuclear layer (mainly rods) to levels that are on average higher, although not statistically significant, than both AAV2/5 and AAV2/9. We additionally found that the ubiquitous cytomegalovirus (CMV), but not the PR-specific GRK1 promoter, transduced pig cones efficiently, presumably because GRK1 is not expressed in pig cones as observed in mice and humans. Indeed, the GRK1 and CMV promoters transduce a similar percentage of murine cones with the CMV reaching the highest expression levels."

According to the news reporters, the research concluded: "Consistent with this, the AAV2/8 vectors with either the CMV or the GRK1 promoter restore cone function in a mouse model of Leber congenital amaurosis type 1 (LCA1), supporting the use of AAV2/8 for gene therapy of LCA1 as well as of other retinal diseases requiring gene transfer to both PR types."

For more information on this research see: Combined Rod and Cone Transduction by Adeno-Associated Virus 2/8. Human Gene Therapy, 2013;24(12):982-992. Human Gene Therapy can be contacted at: Mary Ann Liebert, Inc, 140 Huguenot Street, 3RD Fl, New Rochelle, NY 10801, USA. (Mary Ann Liebert, Inc. -; Human Gene Therapy -

Our news journalists report that additional information may be obtained by contacting A. Manfredi, University of Naples Federico II, Dept. of Translat Med, I-80131 Naples, Italy. Additional authors for this research include E. Marrocco, A. Puppo, G. Cesi, A. Sommella, M. Della Corte, S. Rossi, M. Giunti, C.M. Craft, M.L. Bacci, F. Simonelli, E.M. Surace and A. Auricchio (see also Biotechnology).

Keywords for this news article include: CMV, Biotechnology, Italy, Naples, Europe, Viruses, Genetics, Virology, Gene Therapy, Bioengineering, Cytomegalovirus Infections

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Source: Gene Therapy Weekly

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