Treating Genetic Disorders Using State-Of-The-Art Technology

Abstract

CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR associated Protein 9), basically a bacterial immune system is now widely applicable to engineer genomes of a number of cells and organisms because of its simplicity and robustness. In research avenue the system has been optimized to regulate gene expression, modify epigenome and edit target locus. These applications make CRISPR/Cas9, a technology of choice to edit disease causing mutations as well as the epigenome more efficiently than ever before. Meanwhile its application in in vivo and ex vivo cells is encouraging the scientific community for more vigorous gene therapy and in clinical setups for therapeutic genome editing. Here we review the recent advances that CRISPR-Cas9 mediated genome editing has achieved and is reported in previous studies and address the challenges associated with it.

Frequency of Cystic Fibrosis in Children with Recurrent Respiratory Infections by Sweat Chloride Testing: A Hospital-Based Cross-sectional Study

“…This prevalence of CF in our population therefore demands a population-wise screening program in the form of neonatal IRT analysis. 22 When we compare our prevalence with the United States, CF is found approximately one in 30,000 Americans. 18 Similarly, to our surprise, respiratory tract infection is quite high in our population.…”

Current Applications and Future Perspectives of CRISPR-Cas9 for the Treatment of Lung Cancer

“…Furthermore, its application is encouraging for more vigorous gene therapy in clinical setups. 16 Based on the discovery, there are three main types of CRISPR-Cas9 system (I to III) and with three additional types (IV to VI) being identified more recently. 17 They are different during the processes of immunity, adaptation, expression, and interference, each type acts in distinct mechanisms to ensure genetic manipulation.…”