By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Nutritional and Metabolic Diseases and Conditions have been published. According to news reporting from Houston, Texas, by NewsRx journalists, research stated, "Citrullinemia type 1 (CTLN1) is an autosomal recessive disorder of metabolism caused by a deficiency of argininosuccinate synthetase. Despite optimal management, CTLN1 patients still suffer from lethal metabolic instability and experience life-threatening episodes of acute hyperammonemia."
The news correspondents obtained a quote from the research from the Baylor University College of Medicine, "A murine model of CTLN1 (fold/fold) that displays lethality within the first 21 days of life was used to determine the efficacy of adeno-associated viral (AAV) gene transfer as a potential therapy. An AAV serotype 8 (AAV8) vector was engineered to express the human ASS1 cDNA under the control of a liver-specific promoter (thyroxine-binding globulin, TBG), AAV8-TBG-hASS1, and delivered to 7-10 days old mice via intraperitoneal injection. Greater than 95% of the mice were rescued from lethality and survival was extended beyond 100 days after receiving a single dose of vector. AAV8-TBG-hASS7 treatment resulted in liver-specific expression of hASS1, increased ASS1 enzyme activity, reduction in plasma ammonia and citrulline concentrations and significant phenotypic improvement of the fold/fold growth and skin phenotypes."
According to the news reporters, the research concluded: "These experiments highlight a gene transfer approach using AAV8 vector for liver-targeted gene therapy that could serve as a treatment for CTLN1."
For more information on this research see: Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1. Gene Therapy, 2013;20(12):1188-1191. Gene Therapy can be contacted at: Nature Publishing Group, Macmillan Building, 4 Crinan St, London N1 9XW, England. (Nature Publishing Group - www.nature.com/; Gene Therapy - www.nature.com/gt/)
Our news journalists report that additional information may be obtained by contacting R.J. Chandler, Baylor College of Medicine, Biochem Genet Lab, Houston, TX 77030, United States. Additional authors for this research include T.N. Tarasenko, K. Cusmano-Ozog, Q. Sun, V.R. Sutton, C.P. Venditti and P.J. McGuire (see also Nutritional and Metabolic Diseases and Conditions).
Keywords for this news article include: Biotechnology, Texas, Houston, Viruses, Virology, Treatment, Gene Therapy, United States, Citrullinemia, Bioengineering, Diamino Amino Acids, Adeno-Associated Virus, North and Central America, Nutritional and Metabolic Diseases and Conditions
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