Interviewee: Mario Capecchi.
Mario Capecchi talks about the possibility of correcting genetic defects.
(DNAi Location: Applications > Genes and Medicine > Gene targeting > Possibilities
Cell therapy (somatic))
One is what is called somatic gene therapy, in which what you're doing is altering the gene activity in particular cells. In our body we have two types of cells, we have somatic cells which is all the cells that make our body, except for the germ cells, that is sperm and eggs, and the other is the germ line which are the sperm and eggs. So people are thinking about using this technology to alter, I mean an example would be cystic fibrosis, the main pathology is lung defects, we die, the children die of essentially congestion and infection so, from pneumonia and so on. So one can think about correcting the gene in the lung tissue and in that way relieve that defect. Or similarly if you have a defect in the liver, you would correct the defect in the liver. And this would be very direct and very, and would work because if they have a good gene there instead of the defective gene, everything would be normal.
Gene targeting techniques are used by scientists to simulate human genetic disorders in model organisms. Many scientists believe that gene targeting will lead the way to new methods for correcting genetic defects.