ABSTRACT

Treatment of genetic disease and genetic treatment of disease are two separate matters. The cause of a disease (whether mostly genetic or mostly environmental) and its treatability are quite unconnected. Standard medical treatments that are intended to alleviate disease symptoms-hearing aids or cochlear implants for treating profound deafness, for example-are just as applicable if the disease is mostly genetic or mostly environmental. In this chapter the primary focus is on how genetic technologies are being applied to treat disease, but we begin by taking a broader look at different treatment strategies for genetic disorders.