After watching the video “Genes as Medicine" discuss how gene therapy has been used to treat an inherited form of blindness, and now sickle cell anemia. How were the genes delivered? What model organism was gene therapy tested in? How successful has the treatment been in humans?
After watching the video “Genes as Medicine" discuss how gene therapy has been used to treat an inherited form of blindness, and now sickle cell anemia. How were the genes delivered? What model organism was gene therapy tested in? How successful has the treatment been in humans?
Gene therapy is a medical field that focuses on the utilization of the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease.
A vast majority of the inherited diseases occur due to the faulty protein or absence of a particular protein.
Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease and hereditary diseases in which a defective mutant allele is replaced with a functional one. Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. ... Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell.
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