CRISPR-Cas9 in human Gene Therapy: Clinical Progress, Ethical Dilemmas, and Regulatory Responses

Abstract

The CRISPR-Cas9 technology is a powerful tool for targeted modifications of DNA sequences with unprecedented precision. In enabling researchers with the ability to fix certain lethal and non-lethal mutations that cause diseases like Duchenne muscular dystrophy, sickle cell anaemia, and beta-thalassemia, this powerful technology has shown promise in treatment of these genetic conditions. Several ethical questions have been raised about using CRISPR-Cas9, the most concerning being the editing of human embryos, particularly those pertaining to germline modifications that can alter genes in subsequent generations. Furthermore, the creation of so-called "designer babies" raises ethical questions about consent, autonomy, and potential damage from unintended off-target effects. The possibility that CRISPR-Cas9 may be used for purposes other than medicine, such as altering physical characteristics and intellect, is another concern. The main aim of this review is to curate updated literature on CRISPR-Cas9 technology, its potential for use in genetic therapies, and the ethical and legal issues associated with it. The review also discusses the need for robust ethical framing and regulatory oversight, assuring that gene-editing technologies will be responsible and equitable, with consideration of human dignity and diversity.