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Discovery in HIV may solve efficiency problems for gene therapy
Date:2/14/2013

" with its own genetic material to be effective. After identifying the problem, researchers introduced the element into a lentiviral vector, successfully and significantly improving the quality and quantity of the gene therapy.

Arts and colleagues named the genetic element, Genomic RNA Packaging Enhancer element (or GRPE). During virus production, GRPE coordinates the production and filling of the container with the genetic material of HIV-1, or the desired human gene.

Delivery and success of gene therapy for human cells has the potential of increasing five to ten times with the introduction of the GRPE into the lentiviral vector.

"Using lentivirus for gene transfer appears to be a safe option," said Stanton L. Gerson, MD, director of the Case Comprehensive Cancer Center and the Asa and Patricia Shiverick- Jane Shiverick (Tripp) Professor of Hematological Oncology at Case Western Reserve School of Medicine and director of the Seidman Cancer Center at University Hospitals Case Medical Center, who is not involved in the study. "This discovery could greatly advance the recent successes ongoing in cancer and childhood congenital diseases. Improvements in the technology of gene delivery identified by Arts and his colleagues could lead to many more effective studies that help patients with many different diseases, including cancer. Its impact could be felt in a few short years."

Ultimately, introducing GRPE elements into viral vectors could enhance the ease and effectiveness of gene therapy, which typically uses transplanted human stem cells. Enhanced gene therapy and other improvements in targeted cell delivery might eliminate the need for stem cells and allow cells to be administered directly into patients.


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Contact: Jessica Studeny
jessica.studeny@case.edu
216-368-4692
Case Western Reserve University
Source:Eurekalert

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