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Gene therapy may someday help treat a variety of diseases. Gene therapy, changing a defective gene to a normal one in a living human, shows promise for curing defective genes, but actual successes are rare.
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The goal is to correct or replace the defective genes responsible for disease development. Show more…
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Gene therapy involves the transplantation of normal genes into a genome in place of missing or defective genes. Many scientists believe that gene therapy will lead to revolutionary new treatments for cancer. Why do you think gene therapy is such a promising field for this disease?
Gene therapy aims to replace faulty or disease-causing genes with healthy DNA sequences. How might you use a virus to deliver these new genes into a cell?
Researchers have successfully used gene therapy to ameliorate some human genctic discases by adding a normal gene copy to cells whose genomes originally had only nonfunctional mutant copies of that gene. For example, a form of blindness due to the lack of a single protein called RPE65 has been reversed by introduction of a normal $R P E 65$ gene to cells of the retina of adults. a. The success of this gene therapy approach provides us with clues about the role of the RPE65 protein in the retina. Do you think that RPE65 is needed for the proper development of the human eye? b. Can you see a potential difficulty in applying this gene therapy approach for diseases like microcephaly?
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