Advances in Gene Editing for Treating Inherited Genetic Disorders: A Comprehensive Review

Abstract

Technologies that deal in gene editing have transformed the medical sector especially in the area of curing inherited genetic cases. The fact that one can now accurately edit the genome of an individual has created opportunities in treating ailments that are a result of a given gene mutation. CRISPR-Cas9 has become the most promising tool among the other methods of gene editing because it is precise, thus easy to process, and flexible enough to be used across a variety of platforms. This review article answers all the research questions comprehensively, giving an overview of the current research achievements in gene editing approaches to treat hereditary genetic diseases and focuses specifically on CRISPR-Cas9 and its functions. We discuss the hidden science behind gene editing, the progress of clinical trials and the recent accomplishments using gene editing as a treatment of inherited illnesses such as sickle cell anemia, cystic fibrosis and Duchenne muscular dystrophy. There is also the issues of gene editing and ethical concerns like off-target effects, the mode of delivery and to what extent germ-line editing may be regulated that are included in the paper. Also, we talk about the future of gene editing technologies and how they may become a canonical method of treatment of inherited genetic illnesses. As depicted in this review, although there is promise and challenges to clinical applications of gene editing, this review provides an eye opener into the future of gene therapies in diseases of inherited genetic predisposition. Keywords: gene editing, CRISPR-Cas9, inherited genetic disorders, gene therapy, clinical trials.