Cell and Gene Therapy

Track 15: Human Gene Therapy

Human gene therapy and its function for the treating the human genetic disorders, such as cystic fibrosis, cancer, and other diseases, are considered. Gene therapy is defined as a technique in which a functioning gene is stuck in into a human cell to correct a genetic error or else to introduce a new functioning to the cell. Many methods, including viral vectors and non-viral vectors, have been developed for both ex vivo and in vivo gene transfer into cells. There are several safety and ethical issues related to manipulating the human gene that need to be resolved. Current gene therapy efforts focus on gene insertion into stem cells only. The current human gene project provides the sequences of a vast number of human genes, leading to the identification, characterization, and understanding of genes that are responsible for many human diseases

• Replacing a disease-causing gene with a healthy copy of the gene


• Inactivating a disease-causing gene that is not functioning properly


• Introducing a new or modified gene into the body to help treat a disease