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Scientists Develop Gene Therapy For Effective Treatments For Genetic Disorders And Cancers

by Aruna on September 04, 2009 at 10:14 AM Research News

Scientists in Australia have developed a new gene therapy vector that uses the same machinery as viruses to transport their cargo into cells.

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David Jans, from the Nuclear Signaling Laboratory at Monash University in Victoria, says that this achievement has raised the hope that one day therapeutic DNA will begin to be transferred to a cell's nucleus far more efficiently than in the past, and thus there will be more effective treatments for several 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," he said.

"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," he added.

A gene therapy vector is used to deliver a therapeutic gene or a portion of DNA into a cell nucleus similar to how a syringe is used to inject medicines.

To create the new gene therapy vector, Jans and his colleagues used pieces of different genes to create a protein called a "modular DNA carrier", which can be produced by bacteria.

Writing about their work in The FASEB Journal, the researchers have revealed that this protein carries and delivers therapeutic DNA to a cell's nucleus, where it reprograms a cell to function properly.

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Scientists Develop Gene Therapy For Effective Treatments For Genetic Disorders And Cancers

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