October 08, 2007
Single Shot Gene Therapy Spreads Through Mouse Brain

The brain, not space, is the final frontier. Our brains are all growing old and are the hardest part of the body to repair and rejuvenate. We need gene therapy to do brain rejuvenation. So I'm always happy to come across reports on advances in brain gene therapy. Some researchers have found a way to get really good coverage of gene therapy delivered in to the brains of mice.

By targeting a site in a mouse brain well connected to other areas, researchers successfully delivered a beneficial gene to the entire brain—after one injection of gene therapy. If these results in animals can be realized in people, researchers may have a potential method for gene therapy to treat a host of rare but devastating congenital human neurological disorders, such as Tay-Sachs disease.

Researchers from The Children’s Hospital of Philadelphia and the University of Pennsylvania reported their findings in the September 12 issue of the Journal of Neuroscience.

“After a single injection, this technique succeeded in correcting diseased areas throughout the brain,” said study leader John H. Wolfe, V.M.D., Ph.D., a neurology researcher at The Children’s Hospital of Philadelphia and a professor of pathology and medical genetics at the Penn School of Veterinary Medicine. “This may represent a new strategy for treating genetic diseases of the central nervous system.”

Wolfe and Penn graduate student Cassia N. Cearley performed the study in mice specially bred to have the neurogenetic disease mucopolysaccharidosis type VII (MPS VII). In people, MPS VII, also called Sly syndrome, is a rare, multisystem disease causing mental retardation and death in childhood or early adulthood.

The fact that this gene therapy worked against a lysosomal storage disorder is reason for optimism for brain rejuvenation gene therapies.

Sly syndrome is one of a class of some 60 disorders called lysosomal storage diseases that collectively cause disabilities in about one in 5,000 births. Those diseases account for a significant share of childhood mental retardation and severe, often fatal, disabilities. In each of the lysosomal storage diseases, a defect in a specific gene disrupts the production of an enzyme that cleans up waste products from cells. Cellular debris builds up within cell storage sites called lysosomes, and the waste deposits interfere with basic cell functions. Other examples of lysosomal storage diseases are Tay-Sachs disease, Hunter disease and Pompe disease.

One of Aubrey de Grey's proposed SENS (Strategies for Engineered Negligible Senescence) therapies involves sending genes into cells to enhance lysosomal breakdown of accumulated trash. Basically, use genes from other species to help take out the cellular trash. A successful gene therapy that moves into large numbers of brain cells to enhance lysosomal function would be a step in the right direction for future development of SENS therapies.

Share |      Randall Parker, 2007 October 08 08:05 PM  Biotech Gene Therapy


Comments
Brett Bellmore said at October 9, 2007 3:37 AM:

Of course, one must take into account that, if a single shot could genetically reengineer a volume of human brain comparable to the size of a mouse brain, you'd still be talking a LOT of shots. Mouse brains aren't very large.

I still think we're going to have to use a biological vector which can already travel around the brain, and enter living cells: Intracellular parasites. Kind of icky, I realize, but they've got enormous potential as a genetic engineering vector. Maybe we could persuade Venter and Smith that this would be a good avenue of exploration?

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