By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on Biotechnology. According to news reporting from Iowa City, Iowa, by NewsRx journalists, research stated, "To study safety and efficacy of subretinal adeno-associated virus (AAV) vector AAVBbs1 injection for treatment of a mouse model of Bardet-Biedl syndrome type 1 (BBS1). Constructs containing a wild-type (WT) Bbs1 gene with and without a FLAG tag in AAV2/5 vectors were generated."
The news correspondents obtained a quote from the research from the University of Iowa, "Viral genomes were delivered by subretinal injection to right eyes and sham injections to left eyes at postnatal day 30 (P30) to P60. Transgene expression and BBSome reconstitution were evaluated by immunohistochemistry and Western blotting following sucrose gradient ultracentrifugation. Retinal function was analyzed by electroretinogram (ERG) and structure by optical coherence tomography (OCT). Histology and immunohistochemistry were performed on selected eyes. Expression of FLAG-tagged Bbs1 was demonstrated in photoreceptor cells using antibody directed against the FLAG tag. Coinjection of AAV-GFP demonstrated transduction of 24% to 32% of the retina. Western blotting demonstrated BBS1 protein expression and reconstitution of the BBSome. ERG dark-adapted bright flash b-wave amplitudes were higher in AAV-Bbs1-injected eyes than in sham-injected fellow eyes in more than 50% of 19 animals. Anti-rhodopsin staining demonstrated improved localization of rhodopsin in AAV-Bbs1treated eyes. WT retinas injected with AAV-Bbs1 with or without a FLAG tag showed outer retinal degeneration on ERG, OCT, and histology. In a knock-in model of BBS1, subretinal delivery of AAV-Bbs1 rescues BBSome formation and rhodopsin localization, and shows a trend toward improved ERG."
According to the news reporters, the research concluded: "BBS is challenging to treat with gene therapy due to the stoichiometry of the BBSome protein complex and overexpression toxicity."
For more information on this research see: Subretinal Gene Therapy of Mice With Bardet-Biedl Syndrome Type 1. Investigative Ophthalmology & Visual Science, 2013;54(9):6118-6132. Investigative Ophthalmology & Visual Science can be contacted at: Assoc Research Vision Ophthalmology Inc, 12300 Twinbrook Parkway, Rockville, MD 20852-1606, USA. (The Association for Research in Vision and Ophthalmology - www.arvo.org; Investigative Ophthalmology & Visual Science - www.iovs.org/)
Our news journalists report that additional information may be obtained by contacting S. Seo, University of Iowa, Interdisciplinary Genet Program, Iowa City, IA, United States. Additional authors for this research include R.F. Mullins, A.V. Dumitrescu, S. Bhattarai, D. Gratie, K. Wang, E.M. Stone, V. Sheffield and A.V. Drack (see also technology.html">Biotechnology).
Keywords for this news article include: Biotechnology, Iowa City, Gene Therapy, United States, Bioengineering, Brain Diseases, Bardet-Biedl Syndrome, Hypothalamic Diseases, North and Central America, Central Nervous System Diseases
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