CRISPR-Cas9 Technology in Gene therapy: Scope and Challenges

CRISPR/Cas9 technology has revolutionized the landscape of gene therapy, offering unprecedented precision and versatility in editing specific genes within the human genome.The scope of CRISPR/Cas9 therapeutics now extends to the correction of monogenic disorders like sickle cell disease and thalassemia, targeted cancer therapies, and the treatment of multifactorial conditions such as cardiac diseases, diabetes, and immune disorders.Its application ranges from introducing beneficial mutations, knocking out deleterious genes, to modifying immune cells for enhanced cancer immunotherapy.However, several significant challenges impede the broader clinical adoption of CRISPR/Cas9 gene therapy.Notably, the risks of off-target effects-unintended genetic modifications at nontarget sites-pose critical safety concerns, as they can lead to potentially harmful mutations, including tumorigenesis or cell death