New Gene Therapy Virus as DNA Targetting Tool
Researchers at the University of Washington and Washington University have developed a new gene therapy tool that would allow targeting specific genes in patients' own DNA.
This gene therapy tool is based on the virus that is commonly found in humans, the adeno-associated virus (AAV), and is customized to delivery sequences needed to target the specific gene and to correct genetic defects.
In an article recently published in Nature Biotechnology (Nature Biotechnology - 24, 1022 - 1026), researchers describe how this virus has been modified for targeted gene therapy and demonstrate correcting mutations in two animal models.
The only limitation of this system appears to be the low number of cells that the modified virus could transform, while the advantage is pronounced, given its ability to integrate into the exact location where the genetic mutation exists.
With current technologies using AAV and other viruses for gene therapy, the delivered gene could be inserted randomly into the patients' DNA, in locations that may cause another gene to be affected.
