Neuroscientists have forged an unlikely molecular union as part of their fight against diseases of the brain and nervous system.
The team has brought together the herpes virus and a molecule known as Sleeping Beauty to improve a technology known as gene therapy, which aims to manipulate genes to correct for molecular flaws that cause disease.
The work, detailed in a paper published online in Gene Therapy, has allowed scientists at the University of Rochester Medical Center to reach a long-sought goal: Shuttling into brain cells a relatively large gene that can remain on for an extended period of time.
"We've broken what is in effect a size barrier a limit to how much genetic material we can put into the nucleus of a cell and keep functioning for a long period of time," said neuroscientist William Bowers, Ph.D., a scientist in the Center for Neural Development and Disease and the leader of the team. "That opens up more diseases to possible treatment with gene therapy."
The first author of the paper is Biochemistry graduate student Suresh de Silva, who defends his doctoral thesis later this month.
The molecular rendezvous of Sleeping Beauty and herpes in human brain cells could spell good news in the search for treatments for horrific brain diseases known as pediatric leukodystrophies, or a group of diseases known as lysosomal storage disorders. In many of these diseases, even though just a single gene or protein is defective, the effects are devastating the diseases slowly rob children of their brain cells and are often fatal after years of severe symptoms.
The findings bolster the tools that researchers have when approaching certain diseases, said Bowers, including Usher syndrome, which results in deafness and vision loss; Niemann-Pick disease Type C, a fatal childhood lysosomal storage disorder; and von Willebrand disease, an inherited disease that causes extensive, chronic bleeding.
|Contact: Tom Rickey|
University of Rochester Medical Center