2nd International Conference on
Renaissance London Heathrow Hotel, UK
Gene Therapy is an emerging field in medicine that focuses on treating or preventing diseases by directly targeting and modifying an individual’s genetic material. It involves introducing, removing, or altering genes within a patient’s cells to correct genetic defects, restore normal function, or provide new therapeutic capabilities. Gene therapy shows promise for a wide range of conditions, including monogenic disorders, cancers, cardiovascular diseases, and neurodegenerative disorders. Techniques such as viral and non-viral vector delivery, CRISPR-Cas9 genome editing, and antisense oligonucleotides enable precise modification of genes, addressing the root causes of diseases rather than merely alleviating symptoms. By doing so, gene therapy offers the potential for long-lasting or even permanent treatments. Recent advances in molecular biology, genomics, and clinical research have accelerated the development of gene therapies, with several now FDA-approved. As research continues, gene therapy is revolutionizing personalized and precision medicine, providing new hope for previously untreatable genetic and acquired disorders.
Genetic Engineering in Human Genetics involves manipulating human DNA to prevent, treat, or understand genetic disorders. Key techniques include CRISPR-Cas9, Zinc Finger Nucleases (ZFNs), TALENs, gene therapy, RNA interference, and stem cell engineering, enabling precise modification or replacement of faulty genes. Applications span treating inherited disorders like cystic fibrosis, hemophilia, and sickle cell anemia, cancer therapy through engineered immune cells (CAR-T therapy), and personalized medicine, tailoring treatments to an individual’s genetic profile. Emerging possibilities include germline editing to prevent hereditary diseases, though this raises significant ethical concerns. Challenges include off-target effects, immune responses, long-term safety, and regulatory oversight. Ethical considerations focus on consent, privacy, equitable access, and societal impact. Prospects promise safe correction of monogenic disorders, treatment for complex diseases, and integration of AI in predictive medicine, making genetic engineering a transformative tool in human health and disease management.
