Gene Therapy: Treating Disease by Repairing Genes

From the Introduction:
Although there are thousands of genetic defects that could, in principle, be treated with gene therapy, only a small percentage are considered practical candidates for this type of treatment. Diseases that qualify for gene therapy are debilitating disorders that affect more than 1 percent of the population, the conventional treatments for which are ineffective, costly, or difficult to administer. Many people opt for gene therapy simply because it is their best chance for a normal life, even if they are not completely cured. Gene therapy is a new, potentially dangerous procedure and thus requires careful attention to the selection process

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