This defective copy would have to be removed or inactivated in order to cure these patients.
Gene therapy may also be effective in treating cancer or viral infections such as HIV-AIDS. It can even be used to modify the body's responses to injury. These approaches could be used to reduce scarring after surgery or to reduce restenosis, which is the reclosure of coronary arteries after balloon angioplasty. Each of these cases will be discussed in more detail below, but first we will deal with two technical issues of gene transfer: gene delivery and longevity of gene expression.
Gene Delivery
Whether given as pills or injections, most conventional drugs simply need to reach a minimal level in the bloodstream in order to be effective. In gene therapy, the drug (DNA) must be delivered to the nucleus of a cell in order to function, and a huge number of individual cells must each receive the DNA in order for the treatment to be effective. The situation is further complicated by the fact that a given gene may normally function in only a small portion of the cells in the body, and ectopic expression may be toxic. Thus, successful gene therapy often requires highly efficient delivery of DNA to a very restricted population of cells within the body.
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