Wednesday, May 13, 2020

Genome Editing Essay - 1614 Words

Genome editing of germ line cells using CRISPR/Cas9 system Recent studies suggest that genome editing of germ line cells is an effective strategy for mutated gene correction in sperms and oocytes for the inhibition of onset of inherited disorder. The first time, germ line genome editing in human by CRISPR/Cas9 system , was carried out to correct mutation of HBB (Beta globin) gene in zygote of ÃŽ ²-thalassemia patients by homologous DNA recombination (HDR) [80, 81]. Embryonic genome editing utilized in the treatment of such monogenic diseases prior to birth is a rational use of embryonic genome editing in germ line cells. Homologous DNA recombination by traditional gene editing techniques in mammalian eggs has a low rate (10%) in term of†¦show more content†¦Because PGD usually is carried out by gametes prepared from parents. But parents who is confirmed by PGD to have a high risk of transmition of inherited genetic mutation to their children and want to have their own children, their gametes can be edited directly by Crisper/ca s9 to create healthy children.Nowdays germ line genome editing by programmable nuclease such as Crisper/cas9 is the most useful tool to treat genetic diseases with high risk of inheritance mutation transmition. Unresolved ethical problems still exist and are the main reason for delay onset of clinical trials in this field. Large animal models can be utilized for a variety of monogenic diseases, particularly monogenic neurological disorders like HD and Duchene muscular dystrophy) DMD (. Specificity, efficacy, and side effects of germ line genome editing technology should be assessed by animal models before entering clinical trials. Generating mammalian models of neurodegenerative diseases using CRISPR/Cas9 Cas9 has been well studied. It belongs to the class II CRISPR/Cas system which is the main protein involved in small interfering CRISPR RNA (crRNA). CRISPR RNA often leads to the silencing of invader viruses and plasmids [72, 85]. Several in vitro surveys have shown that Cas9 is guided by gRNA composed of chimeric RNA. Chimeric RNA is composed of crRNA and tracrRNA. They are sufficient toShow MoreRelatedGenome Editing Essay790 Words   |  4 PagesIntroduction Genome editing is a huge leap forward in science and medicine. Because of recent advances in technology, the study of genes and induced ‘point’ mutations have led to the discovery and advancement of methods previously used in order to mutate genes. 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Gene editing used to be a relatively painful and laborious process until the development of CRISPR. In short (and seriously abridging the complex science), CRISPR stands for â€Å"Clustered Regularly-Interspaced Short Palindromic Repeats† and are segments of genetic

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