It sounds straightforward, but the slow rate of progress so far points to hidden problems in getting gene therapy to work efficiently. The first hurdle involves spiriting the new gene through the cell wall. This depends on locating the gene correctly amongst the many other thousands. Genes inserted in the wrong place may not work, and could trigger uncontrolled growth, perhaps even cancer, or turn off a cell altogether. Some of the Issues 1.
Hereditary maladies extract an enormous toll.
Genetic diseases afflict 8,000 newborns in Canada each year. Half of the children in pediatric hospitals are there because of genetic diseases. Operations correcting rarer deficiencies could be restricted to private insurance. Gene therapy can't be used for most of these diseases. It may only be justified for treating some types of heart disease, cancer or diabetes where genes play a dominant role.