By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Hyperammonemia have been presented. According to news reporting from Sydney, Australia, by NewsRx journalists, research stated, "Viral vectors based on adeno-associated virus (AAV) are showing exciting promise in gene therapy trials targeting the adult liver. A major challenge in extending this promise to the pediatric liver is the loss of episomal vector genomes that accompanies hepatocellular proliferation during liver growth."
The news correspondents obtained a quote from the research from the University of Sydney, "Hence maintenance of sufficient transgene expression will be critical for success in infants and children. We therefore set out to explore the therapeutic efficacy and durability of liver-targeted gene transfer in the challenging context of a neonatal lethal urea cycle defect, using the argininosuccinate synthetase deficient mouse. Lethal neonatal hyperammonemia was prevented by prenatal and early postnatal vector delivery; however, hyperammonemia subsequently recurred limiting survival to no more than 33 days despite vector readministration. Antivector antibodies acquired in milk from vector-exposed dams were subsequently shown to be blocking vector readministration, and were avoided by crossfostering vector-treated pups to vector-naive dams. In the absence of passively acquired antivector antibodies, vector redelivery proved efficacious with mice surviving to adulthood without recurrence of significant hyperammonemia."
According to the news reporters, the research concluded: "These data demonstrate the potential of AAV vectors in the developing liver, showing that vector readministration can be used to counter growth-associated loss of transgene expression provided the challenge of antivector humoral immunity is addressed."
For more information on this research see: Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice. Molecular Therapy, 2013;21(10):1823-1831. Molecular Therapy can be contacted at: Nature Publishing Group, 75 Varick St, 9TH Flr, New York, NY 10013-1917, USA. (Elsevier - www.elsevier.com; Molecular Therapy - www.elsevier.com/wps/product/cws_home/622922)
Our news journalists report that additional information may be obtained by contacting C.Y. Kok, University of Sydney, Sch Mol & Microbial Biosci, Sydney, NSW 2006, Australia. Additional authors for this research include S.C. Cunningham, K.H. Carpenter, A.P. Dane, S.M. Siew, G.J. Logan, P.W. Kuchel and I.E. Alexander (see also Hyperammonemia).
Keywords for this news article include: Antibodies, Biotechnology, Sydney, Viruses, Virology, Immunology, Synthetase, Gene Therapy, Bioengineering, Blood Proteins, Hyperammonemia, Immunoglobulins, Enzymes and Coenzymes, Adeno-Associated Virus, Australia and New Zealand
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