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News from NewKerala.com Oct. 9, 1007
Single gene therapy injection may
correct devastating neurological disorders
Washington, October 9 : In what may lead to a new strategy to treat
a host of rare but devastating congenital human neurological
disorders like Tay-Sachs disease, researchers from The Children’s
Hospital of Philadelphia and the University of Pennsylvania have
achieved success in delivering a beneficial gene to the entire brain
of a mouse after one injection of gene therapy.
“After a single injection, this technique succeeded in correcting
diseased areas throughout the brain,” said Dr. John H. Wolfe, 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,” added Dr. Wolfe, who is also the leader
in the study published in the Journal of Neuroscience.
The mice used in the study were specially bred to have the
neurogenetic disease mucopolysaccharidosis type VII (MPS VII), a
rare multi-system disease that causes mental retardation and death
in childhood or early adulthood, which is also known as Sly syndrome.
Dr. Wolfe revealed that he targeted a particular region of the mouse
brain called the ventral tegmental area (VTA), which has numerous
connections with the rest of the brain. He used a neutralized virus
called adeno-associated virus (AAV) as a vector, the delivery
vehicle for the gene that carries coded instructions to produce the
desired enzyme.
“We found that one subtype of AAV was particularly effective for
transporting the gene. The neural pathways carried the virus
throughout the brain, where the gene produced the enzyme. The enzyme
then cleaned up the storage lesions to the point that these storage
lesions were indistinguishable from those found in the brains of
normal mice,” said Wolfe.
He further said that one of the advantages of lysosomal enzymes was
that cells receiving the delivered gene secrete beneficial enzymes
to neighbouring cells, creating a “sphere of correction”.
Dr. Wolfe said that the level of correction resulting from a single
injection was “unprecedented”.
According to him, two millilitres of injected gene therapy might
treat a one-year-old child if the animal results could be
successfully extrapolated to humans.
He, however, has admitted that it may take several years before
direct human treatments may be available.
He also revealed that he was planning further studies to determine
whether his technique would be effective in animals larger than mice.
--- ANI
Fonte:
http://www.newkerala.com/oct.php?action=fullnews&id=9741
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