Why Is Gene Therapy Not Always Effective?

Why does gene therapy not work?

Gene therapies require pumping massive doses of the carrier virus into people–many times more than you’d see in a natural infection.

At such high doses, a virus that doesn’t normally cause an immune response is far less likely to get out of hand and harm the patient..

What is Gene Therapy and why is it not widely successful?

This is only successful if the effects of the disease are reversible or have not resulted in lasting damage to the body. For example, this can be used to treat loss of function disorders such as cystic fibrosis by introducing a functional copy of the gene to correct the disease (see illustration below).

What are the main difficulties in gene therapy?

Difficulties include delivery of the vector to the cell, lack of persistent gene ex- pression in targeted cells, and immune responses to viral gene products, transgenes, or cells targeted by the vectors.

How reliable is gene therapy?

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.

Is Gene Therapy permanent or temporary?

Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene.

Can gene therapy cure all diseases?

Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.