By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Biotechnology have been published. According to news reporting from Chapel Hill, North Carolina, by NewsRx journalists, research stated, "Conceptually, lysosomal storage disorders (LSDs) are highly amenable to gene transfer therapies because they are monogenic and most of them can benefit from cross-correction. However, past gene transfer clinical trials for LSDs, utilizing focal delivery approaches to the brain for Batten disease and Canavan disease, have provided minimal therapeutic benefit to patients."
The news correspondents obtained a quote from the research from the University of North Carolina, "Recent advancements in vector technology now allow for broad central nervous system and/or peripheral organ gene transfer via in vivo and ex vivo approaches, and the application of these technologies has shown considerable promise in small and large animal preclinical studies. Innate and adaptive immune responses pose a risk for the safety and efficacy of gene transfer in some human populations. This review discusses recent gene therapy advancements, other treatment options, and the risks and obstacles associated with the different therapeutic approaches for LSDs. The objective is to provide an introductory summary of where the field of gene therapy currently stands in the treatment of LSDs, and the directions the field is moving toward. Based on results in animal models, global gene transfer has the potential to profoundly alter the course of LSDs."
According to the news reporters, the research concluded: "Moreover, these newer approaches are scalable to large animals and presumably humans."
For more information on this research see: Perspectives In Using Gene Therapy For Lysosomal Storage Diseases. Drugs of the Future, 2013;38(9):635-643. Drugs of the Future can be contacted at: Prous Science, Sau-Thomson Reuters, 398 Provenca, 08025 Barcelona, Spain (see also technology.html">Biotechnology).
Our news journalists report that additional information may be obtained by contacting S.P. Sloniowski, University of North Carolina, Dept. of Ophthalmol, Chapel Hill, NC 27599, United States. Additional authors for this research include J.C. Fox and S.J. Gray.
Keywords for this news article include: Biotechnology, Treatment, Chapel Hill, Gene Therapy, Therapeutics, United States, North Carolina, Bioengineering, Pre-Trial Research, North and Central America, Clinical Trials and Studies
Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2013, NewsRx LLC