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Studies from Auburn University in the Area of Gene Therapy Described (Sustained normalization of neurological disease after intracranial gene therapy...

July 3, 2014



Studies from Auburn University in the Area of Gene Therapy Described (Sustained normalization of neurological disease after intracranial gene therapy in a feline model)

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Research findings on Biotechnology are discussed in a new report. According to news reporting originating in Auburn, Alabama, by NewsRx journalists, research stated, "Progressive debilitating neurological defects characterize feline G(M1) gangliosidosis, a lysosomal storage disease caused by deficiency of lysosomal ?-galactosidase. No effective therapy exists for affected children, who often die before age 5 years."

The news reporters obtained a quote from the research from Auburn University, "An adeno-associated viral vector carrying the therapeutic gene was injected bilaterally into two brain targets (thalamus and deep cerebellar nuclei) of a feline model of G(M1) gangliosidosis. Gene therapy normalized ?-galactosidase activity and storage throughout the brain and spinal cord. The mean survival of 12 treated G(M1) animals was >38 months, compared to 8 months for untreated animals. Seven of the eight treated animals remaining alive demonstrated normalization of disease, with abrogation of many symptoms including gait deficits and postural imbalance."

According to the news reporters, the research concluded: "Sustained correction of the G(M1) gangliosidosis disease phenotype after limited intracranial targeting by gene therapy in a large animal model suggests that this approach may be useful for treating the human version of this lysosomal storage disorder."

For more information on this research see: Sustained normalization of neurological disease after intracranial gene therapy in a feline model. Science Translational Medicine, 2014;6(231):231ra48 (see also Biotechnology).

Our news correspondents report that additional information may be obtained by contacting V.J. McCurdy, Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL 36849, United States. Additional authors for this research include A.K. Johnson, H.L. Gray-Edwards, A.N. Randle, B.L. Brunson, N.E. Morrison, N. Salibi, J.A. Johnson, M. Hwang, R.J. Beyers, S.G. Leroy, S. Maitland, T.S. Denney, N.R. Cox, H.J. Baker, M. Sena-Esteves and D.R Martin.

Keywords for this news article include: Biotechnology, Auburn, Alabama, Gene Therapy, United States, Bioengineering, Galactosidases, Neurological Disease, Enzymes and Coenzymes, North and Central America.

Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC


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Source: Gene Therapy Weekly


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