[tt] Single Shot Gene Therapy Spreads Through Mouse Brain

[Hughes, James J.]

http://www.futurepundit.com/archives/004659.html

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.