CRISPR-Driven Therapeutics for Rare Genetic Disorders

Abstract

Commonness of rare genetic diseases has been estimated at exceeding 300 million individuals everywhere in the globe, however effective therapy to the ailments is uncommon due to genetic heterogeneity, small amount of patients as well as high cost of development. Precision medicine Precision medicine has taken up a new turn with the advent of CRISPRCas genome editing, a technique of targeting and effectively and possibly curatively intervening on DNA. The research article looks at CRISPR mediated applications in the treatment of hereditary disorders that are rare, such as gene repair, gene knock out technology, and transcriptional regulation. Using a translational research paradigm founded on preclinical and initial clinical evidence, the paper evaluates the methods of editing, delivery systems, safety statistics, and clinical progress of CRISPR-based therapies. It has been demonstrated experimentally and clinically that CRISPR therapeutics are more specific and durable and have greater therapeutic potential than the conventional gene therapies. However, there are still concerns of off-target effects, immune reactions, effect delivery as well as regulatory control. The current paper describes a coherent model of CRISPR therapeutic development in rare genetic diseases and offers the future outlook of safe and scalable clinical translation.