What is gene therapy and why hasn't it been successfully developed for the treatment or cure of human genetic diseases to date? What new technique may help advance gene therapy in the future?
What will be an ideal response?
Answer: Gene therapy is the stable introduction of normal, functional copies of genes into people who possess defective or disease-causing genes. It has been difficult to develop successful gene therapies because of the difficulty in finding appropriate vectors that can deliver the normal genes into the target cells. Some viral vectors have had limited success in delivering the desired functional genes, but unfortunately the insertion of the viral DNA into the host-cell genome also dramatically increased the risk of cancer arising in the target cells.
A combination of the CRISPR/Cas system and induced pluripotent stem (iPS) cells could allow scientists to remove stem cells from a patient and edit them with a CRISPR/Cas system. Once edited, iPS cells could be differentiated and reintroduced back into the patient. New vectors, such as adeno-associated virus, are also being explored. Therapies based on RNA, such as RNA-mediated interference are another possibility.
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A) tight junctions; gap junctions B) epidermis; dermis C) dermis; hypodermis D) melanocytes; keratinocytes E) epidermis; hypodermis