Answer to Question #171275 in Genetics for Amir

Question

In an experiment, an animal with a genetic disorder is given gene therapy treatment . For a time, the animal's condition improves, but then a relapse occurs. (a) What has most likely happened ? (b) What must be done to make the improvements in the animal's condition long lasting ?

Answer 1

a) The new genes might have been inserted in the wrong spot in your DNA, there is a chance that the insertion might lead to tumor formation.

b) Insert the functional version of a gene in hopes of replacing the abnormal form. This is used to treat single-gene disorders like hemophilia A and B and cystic fibrosis.