نوع مقاله : مقاله پژوهشی
نویسندگان
1 گروه زیست شناسی، پردیس فرزانگان، دانشگاه سمنان، ایران.
2 گروه ژنتیک پزشکی، دانشکده پزشکی، دانشگاه علوم پزشکی ایران ، تهران، ایران.
3 پردیس فرزانگان دانشگاه سمنان، سمنان، ایران.
4 گروه زیستشناسی، دانشکده علوم پایه، موسسه آموزش عالی آل طه، تهران، ایران
کلیدواژهها
عنوان مقاله English
نویسندگان English
Clustered regularly interspaced short palindromic repeats (CRISPR) were discovered with the discovery of prokaryotic CRISPR-related proteins called Cas, which are capable of creating double-strand breaks in target sequences in the mammalian genome with the help of guide RNA. This discovery led to the development of CRISPR systems that harness natural DNA repair mechanisms to precisely create mutations in defective genes. CRISPR has been used to remove deleterious mutated genes and correct errors in protein-coding sequences to rescue disease phenotypes in preclinical studies and in several clinical trials. However, most genetic disorders result from a combination of other mutations involving deletions and duplications in both coding and non-coding regions of the genome and therefore require complex genome engineering strategies beyond simple gene deletion. To overcome this limitation, the toolbox of natural and engineered CRISPR-Cas systems has expanded dramatically to include a variety of tools. The use of CRISPR technology to edit non-coding genomes, regulate gene expression, make precise genetic changes, and target infectious diseases has the potential to lead to therapeutic solutions for many incurable diseases.
کلیدواژهها English
