In-vivo Gene Therapy - Hope for

a Cure

In-vivo Gene Therapy - Hope for a Cure

Somatic cell gene therapy represents an important advance in medical research which is providing approaches at more effective treatment and even cures for patients. The transfer of genes can be done in two ways. One approach is to remove cells from the patient, infect those cells with a virus containing the gene(s) of interest, and then giving the cells back to the patient. This is referred to as "ex-vivo" (or cellular) gene therapy and was pioneered by Dr. French Anderson and Dr. Michael Blaese at the National Institutes of Health.

In-vivo Gene Therapy - Hope for a Cure

This is proving to be highly successful for some rare genetic diseases as well as several forms of cancer. The second approach is to give the virus (also referred to as the vector), which contains the gene of interest and is modified so it does not cause disease , directly to the patient. This approach is termed "in vivo" gene therapy. Dr. William N. Kelley, Dr. Thomas D. Palella, and Dr. Myron Levine were the first to provide "proof of concept" for this approach in an experimental animal while conducting research at the University of Michigan.

In-vivo Gene Therapy - Hope for a Cure

This approach is showing early promise as a treatment for congenital eye diseases, hemophilia, and hearing loss. The latter application would represent, if successful, the first example of the use of gene therapy to treat a common disease in man . Although Dr. William N. Kelley and his two colleagues are the co-inventors of this "in vivo" approach and the University of Michigan owns the patent for this technique, they extended the use of the patented technology free to non-profit research organizations and investigators and made it available on a non-exclusive license to commercial institutions and laboratories in order not to impede progress in the development of the field .