In-vivo Gene Therapy - Hope for a Cure

1. In-vivo Gene Therapy Hope for a Cure

2. 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.

3. 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.

4. 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 .