If a person's own oligodendrocytes could be made to produce normal ASA, the cells might be able to resume their job of myelinating axons. There are many methods for gene delivery now being tested. Some of these are in the early stages of development. Others are being tested for a variety of genetic diseases. One such method uses a kind of virus called a retrovirus.
Retroviruses are special because they can insert genes into human cells such as oligodendrocytes.
In studies of gene therapy for MLD using retroviruses, researchers take a retrovirus that has been altered so that it can't cause infection and give it a copy of the ASA gene. Then they use the virus to correct the ASA deficiency in oligodendrocytes, which can then be transplanted into the brain. Scientists have begun looking at retrovirus delivery of ASA genes to oligodendrocytes transplanted into rat brains. Researchers at the University of Pittsburgh Medical Center's Pittsburgh Genetics Institute are exploring new approaches to treatment of leukodystrophies under the direction of John A. Barranger, MD, Ph.D. Dr. Barranger joined the University of Pittsburgh in 1990. Working with him are genetic counselors Nadene Henderson and Erin Rice.