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Popular Articles

Aussie And Kiwi Researchers Make Double MS Genetic Discovery
Australian and New Zealand researchers have accelerated research into Multiple Sclerosis by discovering two new locations of genes which will help to unravel the causes of MS and other autoimmune disease.

Increases In Suicides And Murders Linked To Economic Crisis
An article published Online First and in a future edition of The Lancet reports that the rising rates of suicide and murders in the population are directly associated to the growing unemployment rates originated by the economic downturn. Another effect is the decline in road-traffic accidents. Active labor market programs aiming to maintain and reintegrate workers in jobs could tone down some of these unfavorable effects. The article is the work of Dr David Stuckler, of the University of Oxford, UK, and Professor Martin McKee, of the London School of Hygiene and Tropical Medicine, UK, and their team.
News of the day
Advances In Genomic Medicine Will Mean Massive Changes For The NHS It Is Now Time To Prepare For Them - Lords Science Committee
The House of Lords Science and Technology Committee has published a report on Genomic Medicine which argues that recent developments in genomic science stemming from the sequencing of the human genome represent a unique opportunity for real advances in medical care and that the Government and the NHS must take a range of steps to ensure that these advances are realised.
Oncology

Hemophilia A Mice Benefit From Gene Therapy

Hemophilia A is an inherited bleeding disease caused by a lack of the blood clotting protein Factor VIII. It had been hoped that gene therapy would provide a breakthrough in treatment, but the most common gene therapy approach has had little clinical success. However, a team of researchers, at the University of Minnesota Medical School, Minneapolis, has now developed a new approach to target genes specifically to mouse liver sinusoidal endothelial cells (the cells that are the main of Factor VIII) and used it to provide long-term expression of Factor VIII in hemophilia A mice, markedly reducing their disease. They hope that their data might prove to be a step toward successful human clinical trials in individuals with hemophilia A. The team, led by Betsy Kren and Clifford Steer, coated nanoparticles with hyaluron so that they targeted liver sinusoidal endothelial cells. To test the efficacy of gene delivery, the hyaluron-coated nanoparticles were engineered to contain a therapeutic gene (Factor VIII) together with a genetic element known as Sleeping Beauty, which helps the therapeutic gene insert into the genome of the targeted cells (i.e, the liver sinusoidal endothelial cells). Even 50 weeks after hemophilia A mice were injected with these nanoparticles, levels of Factor VIII in the blood were the same as in the blood of normal mice and bleeding times were also similar to those of normal mice. The authors hope that this combination of technologies, the cell-specific nanocapsule delivery system and the Sleeping Beauty genetic element, will prove to be a viable gene therapy. TITLE: Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice http://https://www.the-jci.org/article.php?id=34332 Karen Honey Journal of Clinical Investigation


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