Researchers studying the bleeding disease hemophilia in mice have increased the rodents’ ability to produce a crucial blood-clotting protein, a new study says.
Their success may pave the way for human trials.
Past studies have successfully introduced a gene into animals’ cells to make the clotting protein, Factor VIII, which is missing in people with hemophilia. But attempts at gene therapy in humans have been disappointing, the study authors note.
In the new study, the researchers say they have developed an approach to target genes in mouse liver sinusoidal endothelial cells, which are the main source of Factor VIII.
Researchers hope the new approach will be a step toward successful human clinical trials in individuals with hemophilia A, the most common form of the inherited disorder.
The study by researchers from the University of Minnesota Medical School in Minneapolis is in the June issue of the Journal of Clinical Investigation.
In the study, researchers used nanoparticles coated with hyaluron to target liver sinusoidal endothelial cells. The nanoparticles contained a therapeutic gene (Factor VIII) as well as Sleeping Beauty, a genetic element that helps the therapeutic gene become inserted into the genome of the targeted cells.
Fifty weeks after the treatment, mice injected with these nanoparticles had Factor VIII levels that were the same as those of normal mice. Bleeding times were also similar to those of normal mice.
According to a news release from the journal, it is the authors hope that the combination of Sleeping Beauty and the cell-specific nanocapsule delivery system will result in a successful gene therapy.
The National Heart, Lung and Blood Institute has more on hemophilia.