Gene Therapy

Although great strides have been made in gene therapy in a relatively short time, its potential usefulness has been limited. For instance, it is now known that the vast majority of non-coding regions are no longer considered junk DNA anymore. In fact, these large portions of the genome are involved in the control and regulation of gene expression, and are thus much more complex than originally thought. Even so, each individual cell in the body carries thousands of genes coding for proteins many of which have not yet been identified or characterized.

To address this issue, the National Institutes of Health initiated the Human Genome Project in 1990. The projects 15-year goal was to map the entire human genome. A genome map would help to identify the location of all genes as well as better understand the remaining three billion base pairs. A milestone in the human genome project was completed in 1999 when the first full sequence of an entire chromosome was completed (chromosome 22). The human genome draft sequence was published by HGP and Celera scientists in February 2001 in the journals Science and Nature, respectively.

Some of the genes identified include a gene that predisposes people to obesity, one associated with programmed cell death or apoptosis, a gene that guides HIV viral reproduction, and the genes of inherited disorders like Huntington's disease, Lou Gehrig's disease, and some colon and breast cancers.