Gene Therapy

In 1990, researchers first attempted to combat genetic defects by the transfer of human genes. When a hereditary disorder is the result of a single defective gene, an obvious way to cure the disorder is to add a working copy of the gene. This approach is being used in an attempt to combat cystic fibrosis and it offers potential for treating muscular dystrophy and a variety of other disorders.

One of the first successful attempts was the transfer of a gene encoding the enzyme adenosine deaminase into the bone marrow of two girls suffering from a rare blood disorder caused by the lack of this enzyme. However, while many clinical trials are underway, no others have yet proven successful. This extremely promising approached will require a lot of additional effort.