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New gene therapy vector developed for transferring therapeutic DNA to a cell's nucleus

Published on September 1, 2009 at 3:40 AM · No Comments

New research in the FASEB Journal describes the next generation of gene therapy, raising hope for successful treatment of genetic and other disorders

A research report featured on the cover of the September 2009 print issue of The FASEB Journal (http://www.fasebj.org) describes how Australian scientists developed a new gene therapy vector that uses the same machinery that viruses use to transport their cargo into our cells. As a result of this achievement, therapeutic DNA can be transferred to a cell's nucleus far more efficiently than in the past, raising hopes for more effective treatment of genetic disorders and some types of cancers.

"Through the use of proteins that mimic key functions of viruses for the packaging and transport of therapeutic DNA, we hope to improve the efficiency, and above all, the specificity of human gene therapy," said David Jans, from the Nuclear Signaling Laboratory at Monash University in Victoria, Australia and one of the researchers involved in the work. "Following the creation of efficient, specific and safe DNA delivery vectors, the challenges in human gene therapy will be able to move on from questions of delivery to actual clinical application."

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