Gene therapy involves the delivery of genetic material into cells to treat or prevent disease. It aims to correct genetic defects, introduce therapeutic genes, or modulate gene expression to restore normal function or provide therapeutic benefits. Techniques include viral vectors (like adenoviruses or adeno-
associated viruses) or non-viral methods (such as lipid nanoparticles or electroporation) to deliver genes into target cells. Applications span inherited disorders, cancer, and infectious diseases. Challenges include ensuring safety, efficacy, and long-term expression of therapeutic genes. Ongoing research focuses on refining delivery systems, enhancing specificity, and addressing immune responses to advance the field's clinical potential.
Tracks:
Delivery Systems
Gene Editing Technologies
Immunogenicity and Safety
Regulatory and Ethical Considerations