Gene Therapy Inside Out

Genes make us who we are. Are we tall or short? Do we have blue eyes or brown? Our genes hold the answer. What if part of a gene is defective or missing? Or if a gene changes or mutates? It can cause a disease, like cancer. Enter gene therapy. Among other things, scientists can replace
a bad gene with one that is healthy, add a new gene to perform the function of a defective
or missing gene, or they can turn off a problem gene. How do they do it? Say scientists want to add back a healthy
copy of a gene to cells. They need an efficient way to do it. So scientists use vectors, or vehicles, to
deliver a gene. The healthy gene hitches a ride in the vector. Viruses are one kind of vector. Scientists modify a virus to remove its ability
to cause a disease. The modified virus can then be used to carry
the healthy gene into a cell. Gene therapy can happen on cells inside the
body. Or can happen on cells outsideā€¦ and then
transplanted back in again. Genes make us who we are. And gene therapy holds great promise for a
healthy future.

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