2nd International Conference on
Renaissance London Heathrow Hotel, UK
Gene Editing in Human Genetics refers to a collection of technologies that enable precise modification of DNA sequences to correct genetic defects, study gene function, or develop therapies. Among these, CRISPR-Cas9 is the most widely used system due to its efficiency, accuracy, and ease of design, allowing targeted cutting and modification of DNA. Other technologies include TALENs and Zinc Finger Nucleases (ZFNs), which also enable site-specific DNA editing, and advanced techniques like base and prime editing, which allow single-nucleotide changes without introducing double-strand breaks. Applications in human genetics are vast, including therapeutic correction of monogenic disorders such as sickle cell anemia, cystic fibrosis, and Duchenne muscular dystrophy, as well as research in functional genomics and disease modeling. Preventive applications, such as germline editing, remain highly regulated due to ethical concerns. Key considerations include off-target effects, unintended mutations, safety, equitable access, and societal implications. Regulatory oversight ensures responsible use of these technologies. Gene editing holds transformative potential in medicine and research, offering new avenues for disease treatment, prevention, and understanding human biology, while requiring careful adherence to ethical and safety standards.
