Study Results from Murdoch Children's Research Institute Provide New Insights into Gene Therapy (Correction of Methylmalonic Aciduria In Vivo Using a Codon-Optimized Lentiviral Vector)
By a News Reporter-Staff News Editor at AIDS Weekly -- Investigators publish new report on Biotechnology. According to news reporting from Parkville, Australia, by NewsRx journalists, research stated, "Methylmalonic aciduria is a rare disorder of organic acid metabolism with limited therapeutic options, resulting in high morbidity and mortality. Positive results from combined liver/kidney transplantation suggest, however, that metabolic sink therapy may be efficacious."
The news correspondents obtained a quote from the research from Murdoch Children's Research Institute, "Gene therapy offers a more accessible approach for the treatment of methylmalonic aciduria than organ transplantation. Accordingly, we have evaluated a lentiviral vector-mediated gene transfer approach in an in vivo mouse model of methylmalonic aciduria. A mouse model of methylmalonic aciduria (Mut(-/-)MUT(h2)) was injected intravenously at 8 weeks of age with a lentiviral vector that expressed a codon-optimized human methylmalonyl coenzyme A mutase transgene, HIV-1SDmEF1murSigHutMCM. Untreated Mut(-/-)MUT(h2) and normal mice were used as controls. HIV-1SDmEF1murSigHutMCM-treated mice achieved near-normal weight for age, and Western blot analysis demonstrated significant methylmalonyl coenzyme A enzyme expression in their livers. Normalization of liver methylmalonyl coenzyme A enzyme activity in the treated group was associated with a reduction in plasma and urine methylmalonic acid levels, and a reduction in the hepatic methylmalonic acid concentration."
According to the news reporters, the research concluded: "Administration of the HIV-1SDmEF1murSigHutMCM vector provided significant, although incomplete, biochemical correction of methylmalonic aciduria in a mouse model, suggesting that gene therapy is a potential treatment for this disorder."
For more information on this research see: Correction of Methylmalonic Aciduria In Vivo Using a Codon-Optimized Lentiviral Vector. Human Gene Therapy, 2014;25(6):529-538. 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. - www.liebertpub.com; Human Gene Therapy - www.liebertpub.com/overview/human-gene-therapy-and-part-b-methods/19/)
Our news journalists report that additional information may be obtained by contacting E.S.Y. Wong, Royal Children's Hospital, Murdoch Childrens Res Inst, Parkville, Vic 3052, Australia. Additional authors for this research include C. McIntyre, H.L. Peters, E. Ranieri, D.S. Anson and J.M. Fletcher (see also Biotechnology).
Keywords for this news article include: Parkville, Australia, Australia and New Zealand, Bioengineering, Biotechnology, Gene Therapy, HIV Infections, HIV/AIDS, Primate Lentiviruses, RNA Viruses, Retroviridae, Vertebrate Viruses, Viral Sexually Transmitted Diseases
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