Gene therapy in a pill
Scientists at Northeastern University (Boston, Massachusetts), working under the guidance of Professor Mansoor Amiji, have developed Nanoparticles-in-Microsphere Oral Systems (NiMOS), a safe and effective system for delivering therapeutic genes for oral use that does not require the use of viral vectors.
In experiments on mice, the authors tested the effectiveness of a new method for the treatment of inflammatory bowel disease (IBD) – a collective name for a group of diseases, the most common of which are Crohn's disease and ulcerative colitis. According to statistics, IBD affects about 10 out of 100,000 people in the United States. Existing methods of treating IBD are invasive, have toxic side effects and short-term therapeutic effect.
As a therapeutic gene, scientists used a gene with an anti-inflammatory effect of the cytokine interleukin-10. According to the results obtained, oral administration of the NiMOS system provides transfection of the therapeutic gene and local expression of interleukin-10, which not only reduces the levels of pro-inflammatory cytokines, but also contributes to an increase in body weight, restoration of the length and mass of the large intestine, and also suppresses inflammatory tissue reactions.
As part of the experiment, mice with induced intestinal inflammation were orally injected with interleukin-10 expressing plasmids composed of NiMOS particles and gelatin nanoparticles as a control. Four days after the end of the eight-day course of gene therapy, the body weight of mice that had lost more than 10% of their weight during the illness increased to the initial values. Restoration of the length and mass of the large intestine was also recorded in animals, and the intensity of the inflammatory process decreased markedly.
Despite the positive results of preclinical trials of interleukin-10 gene delivery using viral vectors, the potential toxicity of viruses, especially with repeated use, prevents the introduction of the method into clinical practice.
The NiMOS system, which does not use viral vectors, allows you to bypass this difficult moment and, perhaps, will take its place in the clinic much faster, especially in the treatment of chronic diseases such as inflammatory bowel disease.
The article "Oral IL-10 Gene Delivery in a Microsphere-based Formulation for Local Transfection and Therapeutic Efficacy in Inflammatory Bowel Disease" is published in the May issue of the journal Gene Therapy.
Portal "Eternal youth" www.vechnayamolodost.ru based on the materials of ScienceDaily
26.05.2008