04-06-2013, 12:29 PM
Gene Therapy
Gene Therapy[.ppt (Size: 5.99 MB / Downloads: 248)
What is it?
Gene therapy, which was first conceived in the mid-1970s, aims to insert working copies of genes into cells containing dysfunctional ones
treat, cure, or ultimately prevent disease by changing the expression of a person's genes
current gene therapy is primarily experimental, with most human clinical trials only in the research stages
Most often in context of treating lethal and disabling diseases, potential also for disease prevention
Basic Process of Gene Therapy
Several approaches to gene therapy are being tested, including:
- Replacing a mutated gene that causes disease with a healthy copy of the gene
- Inactivating, or “knocking out,” a mutated gene that is functioning improperly
- Introducing a new gene into the body to help fight a disease
Which Cells Targeted?
somatic gene therapy the recipient's genome is changed, but the change is not passed along to the next generation
germline gene therapy, the sex cells are changed with the goal of passing these changes to their offspring. But, this is not being actively investigated, at least not humans, although a lot of discussion is being conducted about its value and desirability.
Somatic Stem Cells
Somatic stem cells are remarkable cells in the body
These different stem cell types are targets for gene therapy
Genetically treated stem cells, when reintroduced into the patient's body are expected to naturally travel through the blood stream to the bone marrow.
Germline Gene Therapy
Gene therapy in germline cells has the potential to affect not only the individual, but also their children as well.
Any genetic changes in the reproductive cells or changes made to the embryo before the stage of differentiation, would affect all future offspring of that person. This makes an vital distinction, affecting major ethcial issues