Combination of Genetics and Nanotechnology for Down Syndrome Modification: A Potential Hypothesis and Review of the Literature

Tehran University of Medical Sciences Iranian Journal of Public Health 2251-6085 48 3 2019 03 12 Combination of Genetics and Nanotechnology for Down Syndrome Modification: A Potential Hypothesis and Review of the Literature 371 378 Alireza TAFAZOLI Department of Analysis and Bioanalysis of Medicine, Faculty of Pharmacy with the Division of Laboratory Medicine, Medical University of Bialystok, Bialystok, Poland AND Department of Endocrinology, Diabetology and Internal Medicine, Clinical Research Center, Medical University of Bialystok, Bialystok, Poland Farkhondeh BEHJATI Genetics Research Center, University of Social Welfare and Rehabilitation Sciences, Tehran, Iran Dariush D. FARHUD School of Public Health, Tehran University of Medical Sciences, Tehran, Iran AND Department of Basic Sciences, Iranian Academy of Medical Sciences, Tehran, Iran Mohammad Reza ABBASZADEGAN Medical Genetics Research Center, Mashhad University of Medical Sciences, Mashhad, Iran 2019 03 12 Down syndrome (DS) is one of the most prevalent genetic disorders in humans. The use of new approaches in genetic engineering and nanotechnology methods in combination with natural cellular phenomenon can modify the disease in affected people. We consider two CRISPR/Cas9 systems to cut a specific region from short arm of the chromosome 21 (Chr21) and replace it with a novel designed DNA construct, containing the essential genes in chromatin remodeling for inactivating of an extra Chr21. This requires mimicking of the natural cellular pattern for inactivation of the extra X chromosome in females. By means of controlled dosage of an appropriate Nano-carrier (a surface engineered Poly D, L-lactide-co-glycolide (PLGA) for integrating the relevant construct in Trisomy21 brain cell culture media and then in DS mouse model, we would be able to evaluate the modification and the reduction of the active extra Chr21 and in turn reduce substantial adverse effects of the disease, like intellectual disabilities. The hypothesis and study seek new insights in Down syndrome modification. https://ijph.tums.ac.ir/index.php/ijph/article/view/16505 https://ijph.tums.ac.ir/index.php/ijph/article/download/16505/6314