Application of Biotechnology in Diagnosis and Treatment of Human Genetic Disorders

Abstract

Biotechnology is the application of biological molecules to make useful products that can be used to solve problems in different sectors. Medical biotechnology in particular aims to know the root causes of genetic disorders and develop therapeutic strategies. Although human diseases are caused by different factors, those caused by genetic disorders are increasing in prevalent worldwide. Early detection and treatment of these condition is paramount important. Chemical treatments that cure only the symptom of genetic disorders were practiced so far to manage the effect. However, as a result of advances in understanding of biological molecules, treatment of the cause of the disease called gene therapy has been developed more popularity nowadays. Moreover, most research centers abandoned the classical techniques that are prone to error, and began to use high throughput technologies to correct genetic disorders. Among these, CRISPR Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats Associated Cas9) is the most easy to use and allows precise gene editing in higher organisms to repair disease-causing genes by homologous directed repair. The recent development of modified versions CAs9 Nickase that  reduce off target effects and  the “base editor” that preform without double strand DNA break and  without the need to template indicated its application for therapeutic genome editing in man in the future.

Keywords: Biotechnology, CRISPR Cas9, gene therapy, Nickase

DOI: 10.7176/ALST/76-02

Publication date:September 30^th 2019